Study Summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
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Request More InfoInterventions
SRP-9003BIOLOGICAL
Solution for single IV infusion
GlucocorticoidDRUG
Oral tablet (prophylactic)
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| University of California, San Diego-Altman Clinical and Translational Research Institute | La Jolla | California | United States |
| Nationwide Childrens Hospital | Columbus | Ohio | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States |
| Children's Hospital of The King's Daughter | Norfolk | Virginia | United States |
| University Hospital Leuven (UZ Leuven) | Leuven | Vlaams Brabant | Belgium |
| NMRC Gent (UZ Gent) | Ghent | Belgium | |
| Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum | Essen | North Rhine-Westphalia | Germany |
| Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico | Milan | Italy | |
| Hospital Sant Joan de Deu | Barcelona | Spain | |
| Newcastle University | Newcastle upon Tyne | United Kingdom |