2021-7-2 · Duchenne muscular dystrophy is a form of muscular dystrophy worsens quickly. Other muscular dystrophies (including Becker muscular dystrophy) get worse much more slowly.. Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in the muscles). However, it often occurs in people without a known family history of the condition.
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Severe cognitive impairment was found in 33% of patients with DMD. The distribution of mutations in the DMD gene was not uniform, most often the mutations were found in the region from exon 43 to exon 50. Serum concentration of NGF in patients with DMD was higher than in the control group (2391 pg/m …
2014-12-8 · Phase 1 Phase 2. Detailed Description: Part 1: Randomized, placebo-controlled dose-titration to assess safety, tolerability and pharmacokinetics of 4 dose levels of SRP-4053 in genotypically-confirmed DMD patients with deletions amenable to exon 53 skipping. Part 2: Open-label evaluation of SRP-4053 in patients from Part 1, along with newly ...