PTC Therapeutics withdrew a New Drug Application for Translarna, a muscular dystrophy treatment, after the Food and Drug Administration indicated it would likely not grant approval.
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne ...
NMD Pharma A/S, a clinical-stage biotechnology company dedicated to developing novel therapies to restore skeletal muscle health, today announced that top-line data results from its Phase 2a ...
A preclinical study led by the Germans Trias i Pujol Research Institute (IGTP), in collaboration with the Institut de ...
Accelerated approval can serve patients well—but only when surrogate endpoints are reliable and clearly predict outcomes that matter to patients.
Company aligned with FDA on Phase 3 randomized, double-blind, placebo-controlled trial design -- IMPACT DUCHENNE: Company ...
While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...
Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...
Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...
"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...
The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...
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