{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T13:17:15Z","timestamp":1790255835937,"version":"4.1.0"},"reference-count":140,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T00:00:00Z","timestamp":1782864000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,7,1]],"date-time":"2026-07-01T00:00:00Z","timestamp":1782864000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,5,21]],"date-time":"2026-05-21T00:00:00Z","timestamp":1779321600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000780","name":"European Commission","doi-asserted-by":"publisher","award":["101169266"],"award-info":[{"award-number":["101169266"]}],"id":[{"id":"10.13039\/501100000780","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003359","name":"Generalitat Valenciana","doi-asserted-by":"publisher","award":["CIPROM\/2023\/22"],"award-info":[{"award-number":["CIPROM\/2023\/22"]}],"id":[{"id":"10.13039\/501100003359","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Drug Discovery Today"],"published-print":{"date-parts":[[2026,7]]},"DOI":"10.1016\/j.drudis.2026.104706","type":"journal-article","created":{"date-parts":[[2026,5,27]],"date-time":"2026-05-27T23:49:39Z","timestamp":1779925779000},"page":"104706","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":1,"title":["The myotonic dystrophy type 1 drug development pipeline: 2026 Edition"],"prefix":"10.1016","volume":"31","author":[{"given":"Yasmine","family":"Ferchichi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"\u00c0ngela","family":"Natividad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rub\u00e9n","family":"Artero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arturo","family":"L\u00f3pez-Castel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.drudis.2026.104706_b0005","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1038\/s41582-025-01139-x","article-title":"Myotonic dystrophy type 1: clinical diversity, molecular insights and therapeutic perspectives","volume":"21","author":"Rahm","year":"2025","journal-title":"Nat Rev Neurol"},{"key":"10.1016\/j.drudis.2026.104706_b0010","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1212\/CPJ.0000000000000531","article-title":"Consensus-based care recommendations for adults with myotonic dystrophy type 1","volume":"8","author":"Ashizawa","year":"2018","journal-title":"Neurol Clin Pract"},{"key":"10.1016\/j.drudis.2026.104706_b0015","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1159\/000524734","article-title":"Global prevalence of myotonic dystrophy: an updated systematic review and meta-analysis","volume":"56","author":"Liao","year":"2022","journal-title":"Neuroepidemiology"},{"key":"10.1016\/j.drudis.2026.104706_b0020","doi-asserted-by":"crossref","first-page":"e1045","DOI":"10.1212\/WNL.0000000000011425","article-title":"Population-based prevalence of myotonic dystrophy type 1 using genetic analysis of statewide blood screening program","volume":"96","author":"Johnson","year":"2021","journal-title":"Neurology"},{"key":"10.1016\/j.drudis.2026.104706_b0025","doi-asserted-by":"crossref","first-page":"799","DOI":"10.1016\/0092-8674(92)90154-5","article-title":"Molecular basis of myotonic dystrophy: expansion of a trinucleotide (CTG) repeat at the 3\u2032 end of a transcript encoding a protein kinase family member","volume":"68","author":"Brook","year":"1992","journal-title":"Cell"},{"key":"10.1016\/j.drudis.2026.104706_b0030","doi-asserted-by":"crossref","first-page":"1599","DOI":"10.1007\/s10072-024-07826-9","article-title":"Myotonic dystrophies: an update on clinical features, molecular mechanisms, management, and gene therapy","volume":"46","author":"Rimoldi","year":"2025","journal-title":"Neurol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0035","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1093\/emboj\/19.17.4439","article-title":"Recruitment of human muscleblind proteins to (CUG)n expansions associated with myotonic dystrophy","volume":"19","author":"Miller","year":"2000","journal-title":"EMBO J"},{"key":"10.1016\/j.drudis.2026.104706_b0040","doi-asserted-by":"crossref","first-page":"2165","DOI":"10.1093\/hmg\/10.19.2165","article-title":"Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2","volume":"10","author":"Mankodi","year":"2001","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0045","doi-asserted-by":"crossref","DOI":"10.1172\/JCI185426","article-title":"The Splice Index as a prognostic biomarker of strength and function in myotonic dystrophy type 1","volume":"135","author":"Provenzano","year":"2025","journal-title":"J Clin Invest"},{"key":"10.1016\/j.drudis.2026.104706_b0050","doi-asserted-by":"crossref","first-page":"216","DOI":"10.2353\/ajpath.2009.080520","article-title":"Muscleblind-like proteins","volume":"174","author":"Holt","year":"2009","journal-title":"Am J Pathol"},{"key":"10.1016\/j.drudis.2026.104706_b0055","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1016\/j.ajhg.2018.02.010","article-title":"Antisense therapy for a common corneal dystrophy ameliorates TCF4 repeat expansion-mediated toxicity","volume":"102","author":"Zarouchlioti","year":"2018","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0060","doi-asserted-by":"crossref","first-page":"7820","DOI":"10.1074\/jbc.M005960200","article-title":"RNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1","volume":"276","author":"Timchenko","year":"2001","journal-title":"J Biol Chem"},{"key":"10.1016\/j.drudis.2026.104706_b0065","doi-asserted-by":"crossref","first-page":"2802","DOI":"10.1172\/JCI32308","article-title":"Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy","volume":"117","author":"Wang","year":"2007","journal-title":"J Clin Invest"},{"key":"10.1016\/j.drudis.2026.104706_b0070","doi-asserted-by":"crossref","first-page":"2766","DOI":"10.1093\/nar\/29.13.2766","article-title":"In vivo co-localisation of MBNL protein with DMPK expanded-repeat transcripts","volume":"29","author":"Fardaei","year":"2001","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.drudis.2026.104706_b0075","doi-asserted-by":"crossref","first-page":"1285","DOI":"10.1128\/MCB.21.4.1285-1296.2001","article-title":"The CELF family of RNA binding proteins is implicated in cell-specific and developmentally regulated alternative splicing","volume":"21","author":"Ladd","year":"2001","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.drudis.2026.104706_b0080","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1126\/science.280.5364.737","article-title":"Disruption of splicing regulated by a CUG-binding protein in myotonic dystrophy","volume":"280","author":"Philips","year":"1998","journal-title":"Science"},{"key":"10.1016\/j.drudis.2026.104706_b0085","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.molcel.2007.07.027","article-title":"Increased steady state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyper-phosphorylation","volume":"28","author":"Kuyumcu-Martinez","year":"2007","journal-title":"Mol Cell"},{"key":"10.1016\/j.drudis.2026.104706_b0090","doi-asserted-by":"crossref","first-page":"10650","DOI":"10.3390\/ijms241310650","article-title":"Therapeutic targeting of the GSK3\u03b2-CUGBP1 pathway in myotonic dystrophy","volume":"24","author":"Lutz","year":"2023","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0095","doi-asserted-by":"crossref","first-page":"94","DOI":"10.3390\/ijms21010094","article-title":"Correction of RNA-binding protein CUGBP1 and GSK3\u03b2 signaling as therapeutic approach for congenital and adult myotonic dystrophy type 1","volume":"21","author":"Timchenko","year":"2019","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0100","doi-asserted-by":"crossref","DOI":"10.1128\/MCB.00155-19","article-title":"Correction of glycogen synthase kinase 3\u03b2 in myotonic dystrophy 1 reduces the mutant RNA and improves postnatal survival of DMSXL mice","volume":"39","author":"Wang","year":"2019","journal-title":"Mol Cell Biol"},{"key":"10.1016\/j.drudis.2026.104706_b0105","doi-asserted-by":"crossref","first-page":"20333","DOI":"10.1073\/pnas.0809045105","article-title":"A postnatal switch of CELF and MBNL proteins reprograms alternative splicing in the developing heart","volume":"105","author":"Kalsotra","year":"2008","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.drudis.2026.104706_b0110","doi-asserted-by":"crossref","first-page":"858","DOI":"10.1101\/gr.184390.114","article-title":"Antagonistic regulation of mRNA expression and splicing by CELF and MBNL proteins","volume":"25","author":"Wang","year":"2015","journal-title":"Genome Res"},{"key":"10.1016\/j.drudis.2026.104706_b0115","doi-asserted-by":"crossref","first-page":"1978","DOI":"10.1126\/science.1088583","article-title":"A muscleblind knockout model for myotonic dystrophy","volume":"302","author":"Kanadia","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.drudis.2026.104706_b0120","doi-asserted-by":"crossref","first-page":"2087","DOI":"10.1093\/hmg\/ddl132","article-title":"Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy","volume":"15","author":"Lin","year":"2006","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0125","doi-asserted-by":"crossref","first-page":"2482","DOI":"10.1038\/s41467-018-04892-4","article-title":"miR-23b and miR-218 silencing increase muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models","volume":"9","author":"Cerro-Herreros","year":"2018","journal-title":"Nat Commun"},{"key":"10.1016\/j.drudis.2026.104706_b0130","doi-asserted-by":"crossref","DOI":"10.1016\/j.jbc.2026.111219","article-title":"Disruptions of cell signaling pathways in myotonic dystrophy type 1 (DM1) skeletal muscle, their pathogenic impact and potential for combinatorial therapeutics","volume":"302","author":"Ravel-Chapuis","year":"2026","journal-title":"J Biol Chem"},{"key":"10.1016\/j.drudis.2026.104706_b0135","doi-asserted-by":"crossref","first-page":"336","DOI":"10.1016\/j.celrep.2013.12.025","article-title":"The Mef2 transcription network is disrupted in myotonic dystrophy heart tissue, dramatically altering miRNA and mRNA expression","volume":"6","author":"Kalsotra","year":"2014","journal-title":"Cell Rep"},{"key":"10.1016\/j.drudis.2026.104706_b0140","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1186\/s40478-023-01540-x","article-title":"Loss of MBNL1-mediated retrograde BDNF signaling in the myotonic dystrophy brain","volume":"11","author":"Wang","year":"2023","journal-title":"Acta Neuropathol Commun"},{"key":"10.1016\/j.drudis.2026.104706_b0145","doi-asserted-by":"crossref","first-page":"460","DOI":"10.1016\/j.bbadis.2005.12.003","article-title":"Brain-specific change in alternative splicing of Tau exon 6 in myotonic dystrophy type 1","volume":"1762","author":"Leroy","year":"2006","journal-title":"Biochim Biophys Acta"},{"key":"10.1016\/j.drudis.2026.104706_b0150","doi-asserted-by":"crossref","first-page":"57","DOI":"10.3389\/fnmol.2013.00057","article-title":"Brain pathology in myotonic dystrophy: when tauopathy meets spliceopathy and RNAopathy","volume":"6","author":"Caillet-Boudin","year":"2014","journal-title":"Front Mol Neurosci"},{"key":"10.1016\/j.drudis.2026.104706_b0155","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abn2375","article-title":"MECP2-related pathways are dysregulated in a cortical organoid model of myotonic dystrophy","volume":"14","author":"Morelli","year":"2022","journal-title":"Sci Trans Med"},{"key":"10.1016\/j.drudis.2026.104706_b0160","article-title":"288th ENMC International Workshop. Towards better diagnosing, understanding and treating gastrointestinal symptoms in myotonic dystrophy: extended insights and practical recommendations. 16\u201318 May 2025, Hoofddorp, the Netherlands","volume":"56\u201357","author":"Scholten","year":"2025","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.drudis.2026.104706_b0165","doi-asserted-by":"crossref","DOI":"10.1016\/j.drudis.2023.103489","article-title":"The myotonic dystrophy type 1 drug development pipeline","volume":"28","author":"Pascual-Gilabert","year":"2023","journal-title":"Drug Discov Today"},{"key":"10.1016\/j.drudis.2026.104706_b0170","doi-asserted-by":"crossref","DOI":"10.3389\/fddsv.2023.1314077","article-title":"Introduction to small molecule drug discovery and preclinical development","volume":"3","author":"Southey","year":"2023","journal-title":"Front Drug Discov"},{"key":"10.1016\/j.drudis.2026.104706_b0175","doi-asserted-by":"crossref","first-page":"1043","DOI":"10.1016\/j.csbj.2020.04.004","article-title":"Drug repositioning or target repositioning: a structural perspective of drug-target-indication relationship for available repurposed drugs","volume":"18","author":"Parisi","year":"2020","journal-title":"Comput Struct Biotechnol J"},{"key":"10.1016\/j.drudis.2026.104706_b0180","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.tips.2021.01.003","article-title":"Drug repurposing for rare diseases","volume":"42","author":"Roessler","year":"2021","journal-title":"Trends Pharmacol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0185","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/j.arcped.2024.10.009","article-title":"Evaluation of professional practices in the use of mexiletine for the management of childhood myotonia in French pediatric neuromuscular centers (MEXI-PEDI survey)","volume":"32","author":"Barri\u00e8re","year":"2025","journal-title":"Arch Pediatr"},{"key":"10.1016\/j.drudis.2026.104706_b0190","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1016\/j.acvd.2024.03.001","article-title":"Expert opinion on mexiletine treatment in adult patients with myotonic dystrophy","volume":"117","author":"Wahbi","year":"2024","journal-title":"Arch Cardiovasc Dis"},{"key":"10.1016\/j.drudis.2026.104706_b0195","unstructured":"European Medicines Agency (EMA). Namuscla (mexiletine). Accessed 21 January 2026. https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/namuscla; Published 15 January 2026."},{"key":"10.1016\/j.drudis.2026.104706_b0200","doi-asserted-by":"crossref","first-page":"660","DOI":"10.1016\/j.hrthm.2020.11.031","article-title":"To the editor\u2014mexiletine in myotonic dystrophy: beware of ventricular arrhythmias!","volume":"18","author":"Guti\u00e9rrez-Ballesteros","year":"2021","journal-title":"Heart Rhythm"},{"key":"10.1016\/j.drudis.2026.104706_b0205","doi-asserted-by":"crossref","first-page":"1944","DOI":"10.1016\/j.hrthm.2020.05.043","article-title":"Evaluation of mexiletine effect on conduction delay and bradyarrhythmic complications in patients with myotonic dystrophy type 1 over long-term follow-up","volume":"17","author":"Vio","year":"2020","journal-title":"Heart Rhythm"},{"key":"10.1016\/j.drudis.2026.104706_b0210","doi-asserted-by":"crossref","first-page":"1441","DOI":"10.1212\/WNL.0b013e3181dc1a3a","article-title":"Mexiletine is an effective antimyotonia treatment in myotonic dystrophy type 1","volume":"74","author":"Logigian","year":"2010","journal-title":"Neurology"},{"key":"10.1016\/j.drudis.2026.104706_b0215","doi-asserted-by":"crossref","first-page":"e228","DOI":"10.1212\/WNL.0000000000011002","article-title":"Mexiletine in myotonic dystrophy type 1: a randomized, double-blind, placebo-controlled trial","volume":"96","author":"Heatwole","year":"2021","journal-title":"Neurology"},{"key":"10.1016\/j.drudis.2026.104706_b0220","doi-asserted-by":"crossref","first-page":"6260","DOI":"10.18632\/aging.103022","article-title":"Myotonic dystrophy type 1 cells display impaired metabolism and mitochondrial dysfunction that are reversed by metformin","volume":"12","author":"Garc\u00eda-Puga","year":"2020","journal-title":"Aging (Albany NY)"},{"key":"10.1016\/j.drudis.2026.104706_b0225","doi-asserted-by":"crossref","first-page":"e262","DOI":"10.1038\/mtna.2015.35","article-title":"In vitro and in vivo modulation of alternative splicing by the biguanide metformin","volume":"4","author":"Laustriat","year":"2015","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.drudis.2026.104706_b0230","doi-asserted-by":"crossref","first-page":"2855","DOI":"10.1093\/brain\/awy231","article-title":"Improved mobility with metformin in patients with myotonic dystrophy type 1: a randomized controlled trial","volume":"141","author":"Bassez","year":"2018","journal-title":"Brain"},{"key":"10.1016\/j.drudis.2026.104706_b0235","doi-asserted-by":"crossref","first-page":"893","DOI":"10.1074\/jbc.M111.306472","article-title":"Evidence for irreversible inhibition of glycogen synthase kinase-3\u03b2 by tideglusib","volume":"287","author":"Dom\u00ednguez","year":"2012","journal-title":"J Biol Chem"},{"key":"10.1016\/j.drudis.2026.104706_b0240","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.3390\/pharmaceutics17081065","article-title":"Population pharmacokinetics of tideglusib in congenital and childhood myotonic dystrophy type 1: influence of demographic and clinical factors on systemic exposure","volume":"17","author":"Di Deo","year":"2025","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.drudis.2026.104706_b0245","doi-asserted-by":"crossref","first-page":"10760","DOI":"10.3390\/ijms262110760","article-title":"Elevated levels of active GSK3\u03b2 in the blood of patients with myotonic dystrophy type 1 correlate with muscle weakness","volume":"26","author":"Jennings","year":"2025","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0250","doi-asserted-by":"crossref","first-page":"84","DOI":"10.1016\/j.pediatrneurol.2020.08.001","article-title":"A phase 2 study of AMO-02 (tideglusib) in congenital and childhood-onset myotonic dystrophy type 1 (DM1)","volume":"112","author":"Horrigan","year":"2020","journal-title":"Pediatr Neurol"},{"key":"10.1016\/j.drudis.2026.104706_b0255","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1002\/mus.26866","article-title":"Sleep disorders in myotonic dystrophies","volume":"62","author":"Subramony","year":"2020","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.drudis.2026.104706_b0260","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1007\/s40263-020-00703-x","article-title":"Pitolisant: a review in narcolepsy with or without cataplexy","volume":"34","author":"Lamb","year":"2020","journal-title":"CNS Drugs"},{"key":"10.1016\/j.drudis.2026.104706_b0265","doi-asserted-by":"crossref","first-page":"1881","DOI":"10.5664\/jcsm.9328","article-title":"Treatment of central disorders of hypersomnolence: an American Academy of Sleep Medicine clinical practice guideline","volume":"17","author":"Maski","year":"2021","journal-title":"J Clin Sleep Med"},{"key":"10.1016\/j.drudis.2026.104706_b0270","doi-asserted-by":"crossref","first-page":"5350","DOI":"10.3390\/ijms26115350","article-title":"Multisystem symptoms in myotonic dystrophy type 1: a management and therapeutic perspective","volume":"26","author":"Kuntawala","year":"2025","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0275","doi-asserted-by":"crossref","first-page":"1895","DOI":"10.5664\/jcsm.9326","article-title":"Treatment of central disorders of hypersomnolence: an American Academy of Sleep Medicine systematic review, meta-analysis, and GRADE assessment","volume":"17","author":"Maski","year":"2021","journal-title":"J Clin Sleep Med"},{"key":"10.1016\/j.drudis.2026.104706_b0280","unstructured":"Harmony Biosciences. Harmony Biosciences announces positive results from the pivotal bioequivalence study for pitolisant gastro-resistant formulation. Accessed 20 February 2026. https:\/\/ir.harmonybiosciences.com\/news-releases\/news-release-details\/harmony-biosciences-announces-positive-results-pivotal\/; Published 24 November 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0285","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0269683","article-title":"Pharmacotherapy alleviates pathological changes in human direct reprogrammed neuronal cell model of myotonic dystrophy type 1","volume":"17","author":"Eltahir","year":"2022","journal-title":"PLoS One"},{"key":"10.1016\/j.drudis.2026.104706_b0290","doi-asserted-by":"crossref","first-page":"42","DOI":"10.1002\/acn3.271","article-title":"Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy","volume":"3","author":"Nakamori","year":"2016","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.drudis.2026.104706_b0295","article-title":"Erythromycin for myotonic dystrophy type 1: a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial","volume":"67","author":"Nakamori","year":"2023","journal-title":"eClinicalMedicine"},{"key":"10.1016\/j.drudis.2026.104706_b0300","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1021\/acsptsci.9b00020","article-title":"Combination treatment of erythromycin and furamidine provides additive and synergistic rescue of mis-splicing in myotonic dystrophy type 1 models","volume":"2","author":"Jenquin","year":"2019","journal-title":"ACS Pharmacol Transl Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0305","doi-asserted-by":"crossref","first-page":"2500","DOI":"10.1038\/s41598-021-82007-8","article-title":"iPSC-derived cardiomyocytes from patients with myotonic dystrophy type 1 have abnormal ion channel functions and slower conduction velocities","volume":"11","author":"Poulin","year":"2021","journal-title":"Sci Rep"},{"key":"10.1016\/j.drudis.2026.104706_b0310","doi-asserted-by":"crossref","first-page":"943","DOI":"10.3390\/cells11060943","article-title":"Calcium channels in the heart: disease states and drugs","volume":"11","author":"Shah","year":"2022","journal-title":"Cells"},{"key":"10.1016\/j.drudis.2026.104706_b0315","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1038\/nature19102","article-title":"Structural basis for inhibition of a voltage-gated Ca2+ channel by Ca2+ antagonist drugs","volume":"537","author":"Tang","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.drudis.2026.104706_b0320","unstructured":"Design Therapeutics. Our programs\u2014design therapeutics. Accessed 3 February 2026. https:\/\/www.designtx.com\/our-programs\/."},{"key":"10.1016\/j.drudis.2026.104706_b0325","unstructured":"Design Therapeutics. Our approach\u2014how our GeneTAC\u00ae molecules work. Accessed 3 February 2026. https:\/\/www.designtx.com\/our-approach\/."},{"key":"10.1016\/j.drudis.2026.104706_b0330","unstructured":"Design Therapeutics, Inc. Design Therapeutics announces positive preclinical data highlighting disease-modifying potential of its novel DM1 GeneTACs as a treatment for myotonic dystrophy type-1. Accessed 5 February 2026. https:\/\/investors.designtx.com\/news-releases\/news-release-details\/design-therapeutics-announces-positive-preclinical-data\/; Published 8 September 2021."},{"key":"10.1016\/j.drudis.2026.104706_b0335","unstructured":"Design Therapeutics. Design Therapeutics announces plans to initiate patient dosing of DT-818 in myotonic dystrophy type-1 (DM1) in the first half of 2026 and reports third quarter 2025 financial results. Accessed 3 February 2026. https:\/\/investors.designtx.com\/news-releases\/news-release-details\/design-therapeutics-announces-plans-initiate-patient-dosing-dt\/; Published 5 November 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0340","doi-asserted-by":"crossref","first-page":"2249","DOI":"10.1007\/s40120-025-00804-z","article-title":"Assessing the safety and efficacy of lamotrigine as anti-myotonic agent in myotonic dystrophy type 1 (DM1): a longitudinal, open-label, pilot study","volume":"14","author":"Risi","year":"2025","journal-title":"Neurol Ther"},{"key":"10.1016\/j.drudis.2026.104706_b0345","unstructured":"Center for Biologics Evaluation and Research (CBER). What are \u201cbiologics\u201d questions and answers. Food and Drug Administration (FDA). Accessed 21 January 2026. https:\/\/www.fda.gov\/about-fda\/center-biologics-evaluation-and-research-cber\/what-are-biologics-questions-and-answers; Published 21 August 2023."},{"key":"10.1016\/j.drudis.2026.104706_b0350","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1159\/000478077","article-title":"The immunogenicity of biologic therapies","volume":"53","author":"Garc\u00eas","year":"2018","journal-title":"Curr Probl Dermatol"},{"key":"10.1016\/j.drudis.2026.104706_b0355","first-page":"107","article-title":"Biologics: the role of delivery systems in improved therapy","volume":"8","author":"\u0160kalko-Basnet","year":"2014","journal-title":"Biologics"},{"key":"10.1016\/j.drudis.2026.104706_b0360","doi-asserted-by":"crossref","first-page":"299","DOI":"10.5582\/bst.2019.01226","article-title":"Stem cell secretome as a new booster for regenerative medicine","volume":"13","author":"Xia","year":"2019","journal-title":"Biosci Trends"},{"key":"10.1016\/j.drudis.2026.104706_b0365","doi-asserted-by":"crossref","DOI":"10.1016\/j.nmd.2024.07.525","article-title":"Development of a novel fusion protein, JUV-161, that enhances muscle regeneration for treatment of myotonic dystrophy type 1","volume":"43","author":"Kim","year":"2024","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.drudis.2026.104706_b0370","doi-asserted-by":"crossref","first-page":"1973","DOI":"10.1016\/j.biocel.2010.08.010","article-title":"Altered signal transduction pathways and induction of autophagy in human myotonic dystrophy type 1 myoblasts","volume":"42","author":"Beffy","year":"2010","journal-title":"Int J Biochem Cell Biol"},{"key":"10.1016\/j.drudis.2026.104706_b0375","doi-asserted-by":"crossref","first-page":"3091","DOI":"10.3390\/ijms24043091","article-title":"Protein phosphorylation alterations in myotonic dystrophy type 1: a systematic review","volume":"24","author":"Costa","year":"2023","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0380","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102440","article-title":"Nucleic acid therapeutics: past, present, and future","volume":"36","author":"Naeem","year":"2025","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.drudis.2026.104706_b0385","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1038\/s41392-024-02035-4","article-title":"Nucleic acid drugs: recent progress and future perspectives","volume":"9","author":"Sun","year":"2024","journal-title":"Sig Transduct Target Ther"},{"key":"10.1016\/j.drudis.2026.104706_b0390","doi-asserted-by":"crossref","first-page":"7605","DOI":"10.2147\/IJN.S436038","article-title":"A comprehensive review of small interfering RNAs (siRNAs): mechanism, therapeutic targets, and delivery strategies for cancer therapy","volume":"18","author":"Zhang","year":"2023","journal-title":"Int J Nanomed"},{"key":"10.1016\/j.drudis.2026.104706_b0395","unstructured":"Avidity Biosciences. DM1 Pipeline. Accessed 21 January 2026. https:\/\/www.aviditybiosciences.com\/pipeline\/dm1."},{"key":"10.1016\/j.drudis.2026.104706_b0400","doi-asserted-by":"crossref","first-page":"5901","DOI":"10.1093\/nar\/gkad415","article-title":"Targeted tissue delivery of RNA therapeutics using antibody\u2013oligonucleotide conjugates (AOCs)","volume":"51","author":"Malecova","year":"2023","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.drudis.2026.104706_b0405","doi-asserted-by":"crossref","first-page":"763","DOI":"10.1056\/NEJMoa2407326","article-title":"An antibody-oligonucleotide conjugate for myotonic dystrophy type 1","volume":"394","author":"Johnson","year":"2026","journal-title":"N Engl J Med"},{"key":"10.1016\/j.drudis.2026.104706_b0410","unstructured":"Dyne Therapeutics. Our FORCE\u2122 Platform. Accessed 21 January 2026. https:\/\/www.dyne-tx.com\/our-forcetm-platform\/."},{"key":"10.1016\/j.drudis.2026.104706_b0415","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/s43856-025-00733-w","article-title":"FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models","volume":"5","author":"Weeden","year":"2025","journal-title":"Commun Med (Lond)"},{"key":"10.1016\/j.drudis.2026.104706_b0420","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1016\/j.bbadis.2014.05.019","article-title":"Myotonic dystrophies: an update on clinical aspects, genetic, pathology, and molecular pathomechanisms","volume":"1852","author":"Meola","year":"2015","journal-title":"Biochim Biophys Acta"},{"key":"10.1016\/j.drudis.2026.104706_b0425","doi-asserted-by":"crossref","first-page":"862","DOI":"10.1002\/ana.23992","article-title":"Splicing biomarkers of disease severity in myotonic dystrophy","volume":"74","author":"Nakamori","year":"2013","journal-title":"Ann Neurol"},{"key":"10.1016\/j.drudis.2026.104706_b0430","series-title":"Initial Data From the ACHIEVE Trial of DYNE-101 in Adults With Myotonic Dystrophy Type 1 (DM1)","author":"Wolf","year":"2024"},{"key":"10.1016\/j.drudis.2026.104706_b0435","unstructured":"Dyne Therapeutics, Inc. Dyne Therapeutics announces additional one-year clinical data demonstrating functional improvement from phase 1\/2 ACHIEVE trial of zeleciment basivarsen (DYNE-101) for myotonic dystrophy type 1 (DM1). Accessed 21 January 2026. https:\/\/investors.dyne-tx.com\/news-releases\/news-release-details\/dyne-therapeutics-announces-additional-one-year-clinical-data\/; Published 6 October 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0440","unstructured":"Dyne Therapeutics, Inc. Dyne Therapeutics receives FDA Fast Track designation for DYNE-101 for the treatment of myotonic dystrophy type 1. Accessed 21 January 2026. https:\/\/investors.dyne-tx.com\/news-releases\/news-release-details\/dyne-therapeutics-receives-fda-fast-track-designation-dyne-101\/; Published 21 January 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0445","unstructured":"Vertex Pharmaceuticals. R&D Pipeline. Myotonic dystrophy type 1. Accessed 19 February 2026. https:\/\/www.vrtx.com\/our-science\/pipeline\/myotonic-dystrophy-type-1\/."},{"key":"10.1016\/j.drudis.2026.104706_b0450","unstructured":"Entrada Therapeutics. Entrada Therapeutics closes agreement with vertex to discover and develop endosomal escape vehicle-therapeutics for myotonic dystrophy type 1 (DM1). Accessed 22 April 2026. https:\/\/ir.entradatx.com\/news-releases\/news-release-details\/entrada-therapeutics-closes-agreement-vertex-discover-and\/."},{"key":"10.1016\/j.drudis.2026.104706_b0455","doi-asserted-by":"crossref","unstructured":"PepGen. PepGen reports fourth quarter and year-end 2024 financial results and recent corporate highlights. Accessed 19 February 2026. https:\/\/investors.pepgen.com\/news-releases\/news-release-details\/pepgen-reports-fourth-quarter-and-year-end-2024-financial\/; Published 24 February 2025.","DOI":"10.1016\/j.fopow.2025.03.013"},{"key":"10.1016\/j.drudis.2026.104706_b0460","unstructured":"PepGen. PepGen announces IND-enabling preclinical data supporting progression of PGN-EDODM1 into clinical studies. Accessed 5 February 2026. https:\/\/investors.pepgen.com\/news-releases\/news-release-details\/pepgen-announces-ind-enabling-preclinical-data-supporting\/; Published 7 December 2022."},{"key":"10.1016\/j.drudis.2026.104706_b0465","unstructured":"PepGen. PepGen receives U.S. FDA Fast Track designation for PGN-EDODM1 for the treatment of myotonic dystrophy type 1. Accessed 19 February 2026. https:\/\/investors.pepgen.com\/news-releases\/news-release-details\/pepgen-receives-us-fda-fast-track-designation-pgn-edodm1\/; Published 20 February 2024."},{"key":"10.1016\/j.drudis.2026.104706_b0470","unstructured":"PepGen. PepGen announces positive initial results, including robust splicing correction, from ongoing FREEDOM-DM1 trial in patients with DM1. Accessed 19 February 2026. https:\/\/investors.pepgen.com\/news-releases\/news-release-details\/pepgen-announces-positive-initial-results-including-robust\/; Published 24 February 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0475","unstructured":"PepGen. PepGen announces regulatory updates on FREEDOM2. Accessed 19 April 2026. https:\/\/investors.pepgen.com\/news-releases\/news-release-details\/pepgen-announces-regulatory-updates-freedom2\/."},{"key":"10.1016\/j.drudis.2026.104706_b0480","unstructured":"Arrowhead Pharmaceuticals, Inc. Our Pipeline. Accessed 21 January 2026. https:\/\/arrowheadpharma.com\/pipeline\/."},{"key":"10.1016\/j.drudis.2026.104706_b0485","unstructured":"Van Dyke J et al. Silencing DMPK gene by ARO-DM1, an siRNA therapeutic, for type 1 myotonic dystrophy [Poster abstract]. MDA Clinical & Scientific Conference. Accessed 21 January 2026. https:\/\/www.mdaconference.org\/abstract-library\/silencing-dmpk-gene-by-aro-dm1-an-sirna-therapeutic-for-type-1-myotonic-dystrophy\/; 2026."},{"key":"10.1016\/j.drudis.2026.104706_b0490","unstructured":"Arrowhead Pharmaceuticals, Inc. Arrowhead Pharmaceuticals earns $100 million milestone from Sarepta Therapeutics. July 2025. Accessed 21 January 2026. https:\/\/arrowheadpharma.com\/news-press\/arrowhead-pharmaceuticals-earns-100-million-milestone-from-sarepta-therapeutics\/."},{"key":"10.1016\/j.drudis.2026.104706_b0495","unstructured":"Arrowhead Pharmaceuticals, Inc. Science & Innovation | Arrowhead Pharmaceuticals. Accessed 11 February 2026. https:\/\/arrowheadpharma.com\/science-and-innovation\/."},{"key":"10.1016\/j.drudis.2026.104706_b0500","unstructured":"Sarepta Therapeutics. Sarepta announces first clinical data from siRNA pipeline targeting FSHD1 and DM1. Accessed 19 April 2026. https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-announces-first-clinical-data-sirna-pipeline-targeting."},{"key":"10.1016\/j.drudis.2026.104706_b0505","doi-asserted-by":"crossref","first-page":"5933","DOI":"10.1016\/j.ymthe.2025.10.063","article-title":"AAV-mediated DMPK silencing: a defining moment in myotonic dystrophy type 1 therapeutics","volume":"33","author":"Chahine","year":"2025","journal-title":"Mol Ther"},{"key":"10.1016\/j.drudis.2026.104706_b0510","doi-asserted-by":"crossref","first-page":"6350","DOI":"10.1016\/j.ymthe.2025.08.050","article-title":"Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1","volume":"33","author":"Tomassy","year":"2025","journal-title":"Mol Ther"},{"key":"10.1016\/j.drudis.2026.104706_b0515","unstructured":"Sanofi Sanofi\u2019s SAR446268 earns US Fast Track designation for the treatment of non-congenital myotonic dystrophy type 1. Accessed 22 November 2025. https:\/\/www.sanofi.com\/en\/media-room\/press-releases\/2025\/2025-09-23-05-00-00-3154388; Published 23 September 2025."},{"key":"10.1016\/j.drudis.2026.104706_b0520","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1016\/j.omtn.2020.07.021","article-title":"Therapeutic potential of antagomiR-23b for treating myotonic dystrophy","volume":"21","author":"Cerro-Herreros","year":"2020","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.drudis.2026.104706_b0525","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.adn6525","article-title":"AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action","volume":"10","author":"Cerro-Herreros","year":"2024","journal-title":"Sci Adv"},{"key":"10.1016\/j.drudis.2026.104706_b0530","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1016\/j.ajhg.2026.01.016","article-title":"Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1","volume":"113","author":"Gonz\u00e1lez-Mart\u00ednez","year":"2026","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0535","unstructured":"ARTHEx Biotech. ARTHEx Biotech announces first patient dosed in phase I-IIa ArthemiR\u2122 trial for myotonic dystrophy type 1 (DM1). Accessed 21 January 2026. https:\/\/www.arthexbiotech.com\/post\/arthex-biotech-announces-first-patient-dosed-in-phase-i-iia-arthemir-tm-trial-for-myotonic-dystrophy-type-1-dm1; Published 15 January 2024."},{"key":"10.1016\/j.drudis.2026.104706_b0540","article-title":"Fatty acid mediated oligonucleotide uptake system enables antimiR-23b mediated functional and molecular rescue of cognitive dysfunction in DMSXL mice","author":"Piqueras-Losilla","year":"2025","journal-title":"Arthex Biotech"},{"key":"10.1016\/j.drudis.2026.104706_b0545","unstructured":"ARTHEx Biotech. ARTHEx Biotech announces ATX-01 has been granted Orphan Drug Designation by the FDA. Accessed 21 January 2026. https:\/\/www.arthexbiotech.com\/post\/arthex-biotech-announces-atx-01-has-been-granted-orphan-drug-designation-by-the-fda; Published 20 July 2022."},{"key":"10.1016\/j.drudis.2026.104706_b0550","unstructured":"ARTHEx Biotech. ARTHEx Biotech announces FDA Rare Pediatric Designation (RPD) of ATX-01 for the treatment of myotonic dystrophy type 1 (DM1). Accessed 21 January 2026. https:\/\/www.arthexbiotech.com\/post\/arthex-biotech-announces-fda-rare-pediatric-designation-rpd-of-atx-01-for-the-treatment-of-myotonic-dystrophy-type-1-dm1; Published 7 November 2024."},{"key":"10.1016\/j.drudis.2026.104706_b0555","unstructured":"ARTHEx Biotech. Arthex Biotech granted FDA Fast Track designation for ATX-01 for the treatment of myotonic dystrophy type 1 (DM1). Accessed 19 April 2026. https:\/\/www.arthexbiotech.com\/post\/arthex-biotech-granted-fda-fast-track-designation-for-atx-01-for-the-treatment-of-myotonic-dystrophy-type-1-dm1."},{"key":"10.1016\/j.drudis.2026.104706_b0560","unstructured":"Arrakis Therapeutics. Myotonic dystrophy type 1\u2014Pipeline Arrakis Therapeutics. December 2025. Accessed 3 February 2026. https:\/\/arrakistx.com\/myotonic-dystrophy-type-1\/."},{"key":"10.1016\/j.drudis.2026.104706_b0565","unstructured":"Arrakis Therapeutics. Arrakis Therapeutics unveils data for its lead, rationally designed, RNA-targeted small molecule (rSM) drug program demonstrating disease-modifying binding to RNA to treat myotonic dystrophy. December 2024. Accessed 3 February 2026. https:\/\/arrakistx.com\/press-release\/arrakis-therapeutics-unveils-data-for-its-lead-rationally-designed-rna-targeted-small-molecule-rsm-drug-program-demonstrating-disease-modifying-binding-to-rna-to-treat-myotonic-dystrophy\/."},{"key":"10.1016\/j.drudis.2026.104706_b0570","doi-asserted-by":"crossref","first-page":"1165","DOI":"10.1016\/j.ajhg.2024.04.015","article-title":"Therapeutic validation of MMR-associated genetic modifiers in a human ex vivo model of Huntington disease","volume":"111","author":"Ferguson","year":"2024","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0575","doi-asserted-by":"crossref","DOI":"10.1016\/j.dnarep.2025.103817","article-title":"Therapeutic targeting of mismatch repair proteins in triplet repeat expansion diseases","volume":"147","author":"Marzec","year":"2025","journal-title":"DNA Repair"},{"key":"10.1016\/j.drudis.2026.104706_b0580","unstructured":"LoQus23 Therapeutics. Science. Accessed 3 February 2026. https:\/\/loqus23.com\/science\/."},{"key":"10.1016\/j.drudis.2026.104706_b0585","unstructured":"LoQus23 Therapeutics. LoQus23 Therapeutics nominates LQT-23 as first-in-class, oral drug candidate for Huntington\u2019s disease. January 2026. Accessed 3 February 2026. https:\/\/loqus23.com\/loqus23-therapeutics-nominates-lqt-23-as-first-in-class-oral-drug-candidate-for-huntingtons-disease\/."},{"key":"10.1016\/j.drudis.2026.104706_b0590","unstructured":"Rgenta Therapeutics. Our Priority Development Pipeline | Rgenta Therapeutics. Accessed 5 February 2026. https:\/\/www.rgentatx.com\/our-pipeline."},{"key":"10.1016\/j.drudis.2026.104706_b0595","doi-asserted-by":"crossref","first-page":"650","DOI":"10.1093\/hmg\/ddy375","article-title":"FAN1 modifies Huntington\u2019s disease progression by stabilizing the expanded HTT CAG repeat","volume":"28","author":"Goold","year":"2019","journal-title":"Hum Mol Genet"},{"key":"10.1016\/j.drudis.2026.104706_b0600","doi-asserted-by":"crossref","first-page":"9445","DOI":"10.1038\/s41467-025-64485-w","article-title":"Mechanism of trinucleotide repeat expansion by MutS\u03b2-MutL\u03b3 and contraction by FAN1","volume":"16","author":"Senoussi","year":"2025","journal-title":"Nat Commun"},{"key":"10.1016\/j.drudis.2026.104706_b0605","unstructured":"EditForce. Platform. \u30a8\u30c7\u30a3\u30c3\u30c8\u30d5\u30a9\u30fc\u30b9\u682a\u5f0f\u4f1a\u793e. Accessed 19 February 2026. https:\/\/www.editforce.co.jp\/en\/technology\/."},{"key":"10.1016\/j.drudis.2026.104706_b0610","unstructured":"EditForce Pipeline. Accessed 3 February 2026. https:\/\/www.editforce.co.jp\/en\/business\/."},{"key":"10.1016\/j.drudis.2026.104706_b0615","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.adq2005","article-title":"Pentatricopeptide repeat protein targeting CUG repeat RNA ameliorates RNA toxicity in a myotonic dystrophy type 1 mouse model","volume":"17","author":"Imai","year":"2025","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.drudis.2026.104706_b0620","unstructured":"Modalis Therapeutics. Pipeline. Accessed 5 February 2026. https:\/\/modalistx.com\/en\/business\/pipeline\/."},{"key":"10.1016\/j.drudis.2026.104706_b0625","unstructured":"Modalis Therapeutics. CRISPR-GNDM technology. Accessed 5 February 2026. https:\/\/modalistx.com\/en\/science\/crispr-gndm\/."},{"key":"10.1016\/j.drudis.2026.104706_b0630","doi-asserted-by":"crossref","first-page":"4385","DOI":"10.3390\/ijms20184385","article-title":"An overview of circular RNAs and their implications in myotonic dystrophy","volume":"20","author":"Czubak","year":"2019","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.drudis.2026.104706_b0635","unstructured":"noctuRNA Therapeutics. Pipeline. Accessed 4 February 2026. https:\/\/nocturnatx.com\/pipeline\/."},{"key":"10.1016\/j.drudis.2026.104706_b0640","unstructured":"Kinea Bio. Pipeline. Accessed 20 February 2026. https:\/\/kineabio.com\/pipeline."},{"key":"10.1016\/j.drudis.2026.104706_b0645","unstructured":"Kinea Bio. Science. Accessed 20 February 2026. https:\/\/kineabio.com\/science."},{"key":"10.1016\/j.drudis.2026.104706_b0650","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1038\/s41551-021-00838-2","article-title":"Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats","volume":"6","author":"Arandel","year":"2022","journal-title":"Nat Biomed Eng"},{"key":"10.1016\/j.drudis.2026.104706_b0655","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0065414","article-title":"Efficacy of muscle exercise in patients with muscular dystrophy: a systematic review showing a missed opportunity to improve outcomes","volume":"8","author":"Gianola","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.drudis.2026.104706_b0660","article-title":"Strength training and aerobic exercise training for muscle disease","volume":"12","author":"Voet","year":"2019","journal-title":"Cochrane Database Syst Rev"},{"key":"10.1016\/j.drudis.2026.104706_b0665","doi-asserted-by":"crossref","DOI":"10.1172\/JCI156125","article-title":"Aerobic exercise elicits clinical adaptations in myotonic dystrophy type 1 patients independently of pathophysiological changes","volume":"132","author":"Mikhail","year":"2022","journal-title":"J Clin Invest"},{"key":"10.1016\/j.drudis.2026.104706_b0670","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/j.nmd.2024.05.009","article-title":"Resistance training in women with myotonic dystrophy type 1: a multisystemic therapeutic avenue","volume":"40","author":"Girard-C\u00f4t\u00e9","year":"2024","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.drudis.2026.104706_b0675","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.3233\/JND-230099","article-title":"Resistance exercise training rescues mitochondrial dysfunction in skeletal muscle of patients with myotonic dystrophy type 1","volume":"10","author":"Di Leo","year":"2023","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.drudis.2026.104706_b0680","doi-asserted-by":"crossref","DOI":"10.1111\/apha.70135","article-title":"A 12-week strength training improves mitochondrial respiration, H2O2 emission and skeletal muscle integrity in women with myotonic dystrophy type 1","volume":"241","author":"Marcangeli","year":"2025","journal-title":"Acta Physiol (Oxf)"},{"key":"10.1016\/j.drudis.2026.104706_b0685","doi-asserted-by":"crossref","first-page":"1794","DOI":"10.3390\/ijerph18041794","article-title":"Metabolic alterations in myotonic dystrophy type 1 and their correlation with lipin","volume":"18","author":"Mateus","year":"2021","journal-title":"Int J Environ Res Public Health"},{"key":"10.1016\/j.drudis.2026.104706_b0690","doi-asserted-by":"crossref","first-page":"2097","DOI":"10.1016\/j.csbj.2024.05.009","article-title":"Disturbance of the human gut microbiota in patients with myotonic dystrophy type 1","volume":"23","author":"Mahdavi","year":"2024","journal-title":"Comput Struct Biotechnol J"},{"key":"10.1016\/j.drudis.2026.104706_b0695","doi-asserted-by":"crossref","first-page":"733","DOI":"10.1016\/j.csbj.2025.02.016","article-title":"Influence of CTG repeats from the human DM1 locus on murine gut microbiota","volume":"27","author":"Mahdavi","year":"2025","journal-title":"Comput Struct Biotechnol J"},{"key":"10.1016\/j.drudis.2026.104706_b0700","doi-asserted-by":"crossref","first-page":"390","DOI":"10.1017\/neu.2024.27","article-title":"A meta-analysis of the prevalence of neuropsychiatric disorders and their association with disease onset in myotonic dystrophy","volume":"36","author":"Pascual-Morena","year":"2024","journal-title":"Acta Neuropsychiatr"}],"container-title":["Drug Discovery Today"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S135964462600111X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S135964462600111X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T20:39:40Z","timestamp":1789504780000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S135964462600111X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,7]]},"references-count":140,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2026,7]]}},"alternative-id":["S135964462600111X"],"URL":"https:\/\/doi.org\/10.1016\/j.drudis.2026.104706","relation":{},"ISSN":["1359-6446"],"issn-type":[{"value":"1359-6446","type":"print"}],"subject":[],"published":{"date-parts":[[2026,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The myotonic dystrophy type 1 drug development pipeline: 2026 Edition","name":"articletitle","label":"Article Title"},{"value":"Drug Discovery Today","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.drudis.2026.104706","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Ltd.","name":"copyright","label":"Copyright"}],"article-number":"104706"}}