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A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

purpose:

The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids

sponsor: Santhera Pharmaceuticals

location: United States, Austria, Belgium, Bulgaria, France, Germany, Hungary, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

sponsor: Sarepta Therapeutics

location: United States

A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy

purpose: Current DMD therapies are aimed at increasing dystrophin levels and reducing inflammation. Improved anti-inflammatory therapies are needed to safely treat this pathology and delay disease progression. This study will be conducted in a single-centre and assess the safety, efficacy and PK of ATL1102 in non-ambulatory patients with DMD. Read more:...

sponsor: Antisense Therapeutics Limited

location: Australia

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

purpose:

This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and two validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.

sponsor: PTC Therapeutics

location: United States

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

purpose:

This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for greater than or equal to (>=) 9 months.

The study will have a single visit (Visit 1).

sponsor: PTC Therapeutics

location: United States

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy

purpose:

This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD). Research: Anti-inflammatory

sponsor: Mallinckrodt

location: United States, Bulgaria, Israel, Italy, Mexico, Serbia, Spain, Turkey

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