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The degree of improvement in muscle strength and respiratory health varies with SMA genotype, severity of baseline neuromuscular and pulmonary impairment, medication used, and timing of the first dose. A spectrum of pulmonary outcomes has been reported with these novel medications when used early and in conjunction with proactive multidisciplinary management of comorbidities. In this review, we summarize the reported impact of these novel therapies on pulmonary well\u2010being and the improving trajectory of pulmonary morbidity, compared to the natural history of SMA. The importance of ongoing clinical monitoring albeit the improved phenotype is reiterated. We also discuss the limitations of the current SMA\u2010therapy trials and offer suggestions for future clinical\u2010outcome studies and long\u2010term monitoring.<\/jats:p>","DOI":"10.1002\/ppul.25135","type":"journal-article","created":{"date-parts":[[2020,10,24]],"date-time":"2020-10-24T14:26:17Z","timestamp":1603549577000},"page":"721-728","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":30,"title":["The respiratory impact of novel therapies for spinal muscular atrophy"],"prefix":"10.1002","volume":"56","author":[{"given":"Grace R.","family":"Paul","sequence":"first","affiliation":[{"name":"Department of Pediatric Pulmonary Medicine Nationwide Children's Hospital and The Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney","family":"Gushue","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pulmonary Medicine Oregon Health Sciences University Portland Oregon USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kavitha","family":"Kotha","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pulmonary Medicine Nationwide Children's Hospital and The Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4938-9075","authenticated-orcid":false,"given":"Richard","family":"Shell","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pulmonary Medicine Nationwide Children's Hospital and The Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,11,2]]},"reference":[{"key":"e_1_2_10_2_1","volume-title":"Respiratory Physiology\u2010The Essentials","author":"West JB","year":"2020"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/resp.12330"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.prrv.2018.07.002"},{"key":"e_1_2_10_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.24497"},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0165818"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.201906-1175LE"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1136\/thoraxjnl-2012-201964"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21093297"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1080\/14656566.2019.1704732"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9072222"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)31408-8"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2017-317412"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006281"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-180315"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25533"},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2019.09.007"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/ppul.24203"},{"key":"e_1_2_10_25_1","doi-asserted-by":"crossref","unstructured":"DayJW ChiribogaCA CrawfordTO et al.Onasemnogene abeparvovec\u2010xioi gene\u2010replacement therapy for spinal muscular atrophy type 1 (SMA1): phase 3 US study (STR1VE) update (1828). 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