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Kindergeneeskunde

Klinische studies Duchenne muscular dystrophy

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Duchenne Muscular Dystrophy (DMD) is an X-linked progressive muscular disorder caused by mutations in the dystrophin gene, affecting up to 1 in 5000 boys. Mutations within this gene result in a lack of expression of the functional form of its major protein product, dystrophin, causing damage to the muscle fibers, resulting in progressive muscle weakness and atrophy. The latter is responsible for the clinical manifestations of decreased physical functionality, respiratory and cardiac failure, and eventually death.

DYNE251-DMD-201

A randomized, double-blind, placebo-controlled, multiple ascending dose study assessing safety, tolerability, pharmacodynamics, efficacy, and pharmacokinetics of dyne-251 administered to participants with duchenne muscular dystrophy amenable to exon 51 skipping

C3391003

A phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pf-06939926 for the treatment of duchenne muscular dystrophy.

Sarepta 5051-201

A phase 2, two-part, multiple-ascending-dose study of srp-5051 for dose determination, then dose expansion, in patients with duchenne muscular dystrophy amenable to exon 51-skipping treatment.

Lelantos

A phase 3, randomized, double-blind, trial of pamrevlumab (fg-3019) or placebo in combination with systemic corticosteroids in subjects with non-ambulatory duchenne muscular dystrophy (dmd).

Essence-301

A double-blind, placebo-controlled, multicenter study with an open-label extension to evaluate the efficacy and safety of SRP-4045 and SRP-4053 in patients with duchenne muscular dystrophy.

Givinostat 2

Open label, long-term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies.

Laatste aanpassing: 27 juni 2023