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Treatment options, such as enzyme replacement therapy (ERT), substrate replacement therapy, and pharmacological chaperone therapy, are available for several LSDs, including Gaucher disease (GD), Fabry disease (FD), and Hunter syndrome (mucopolysaccharidosis type II [MPS II]). However, patients in some countries face challenges accessing treatments owing to limited availability of locally licensed, approved drugs.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The Takeda LSD Charitable access program aims to meet the needs of individuals with GD, FD or MPS II with the greatest overall likelihood of benefit, in selected countries, through donation of ERT to nonprofit organizations, and support for medical capacity-building as well as family support via independent grants. Long-term aims of the program are to establish sustainable healthcare services delivered by local healthcare providers for patients with rare metabolic diseases. Patients receiving treatment through the program are monitored regularly, and their clinical data and progress are reviewed annually by an independent medical expert committee (MEC). The MEC also selects patients for enrollment completely independent from the sponsoring company.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>As of 31 August, 2019, 199 patients from 13 countries were enrolled in the program; 142 with GD, 41 with MPS II, and 16 with FD. Physicians reported improvements in clinical condition for 147 (95%) of 155 patients with follow-up data at 1\u00a0year.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The response rate for follow-up data at 1\u00a0year was high, with data collected for\u2009&gt;\u200990% of patients who received ERT through the program showing clinical improvements in the majority of patients. These findings suggest that the program can benefit selected patients previously unable to access disease-specific treatments. Further innovative solutions and efforts are needed to address the challenges and unmet needs of patients with LSDs and other rare diseases around the world.<\/jats:p><\/jats:sec>","DOI":"10.1186\/s13023-020-01645-9","type":"journal-article","created":{"date-parts":[[2021,1,6]],"date-time":"2021-01-06T18:03:54Z","timestamp":1609956234000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["A charitable access program for patients with lysosomal storage disorders in underserved communities worldwide"],"prefix":"10.1186","volume":"16","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6474-9959","authenticated-orcid":false,"given":"Atul","family":"Mehta","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Uma","family":"Ramaswami","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joseph","family":"Muenzer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roberto","family":"Giugliani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kurt","family":"Ullrich","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tanya","family":"Collin-Histed","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zoya","family":"Panahloo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hartmann","family":"Wellhoefer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joel","family":"Frader","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2021,1,6]]},"reference":[{"key":"1645_CR1","volume-title":"Williams hematology","author":"A Zimran","year":"2016","unstructured":"Zimran A, Elstein D. 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UR has served on advisory boards for Amicus, Chiesi, Idorsia, Shire*, and Sanofi-Genzyme; has received honoraria from Amicus and Shire*; and received travel expenses from Amicus, Sanofi-Genzyme, and Shire*. JM has served on advisory boards for BioMarin, Denali Therapeutics, Green Cross, Regenxbio, Sanofi-Genzyme, and Shire*; and has received consulting fees from BioMarin, Denali Therapeutics, Eloxx, Green Cross, PTC Therapeutics, Regenxbio, Sangamo, Sanofi-Genzyme, and Shire*. RG has served on advisory boards for Amicus, BioMarin, GC Pharma, Inventiva, JCR Pharmaceuticals, Lysogene, Regenxbio, Sanofi-Genzyme, Shire*, Sobi, and Ultragenyx; has received research grants from Amicus, Armagen, BioMarin, GC Pharma, JCR Pharmaceuticals, Lysogene, Regenxbio, Sanofi-Genzyme, and Shire*; has received honoraria from Amicus; and has received travel expenses from BioMarin, JCR Pharmaceuticals, Sanofi-Genzyme, and Shire*. KU and TC-H have nothing to disclose. ZP and HW were employees of Shire International GmbH* at the time the study took place. JF has received consulting fees from Dicerna Pharmaceuticals and travel expenses from Shire*. *A Takeda company.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"8"}}