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Phase I clinical trials started in 2004. [16] In 2010, PTC Therapeutics released preliminary results of its phase IIb clinical trial for Duchenne muscular dystrophy, with participants not showing a significant improvement in the six minute walk distance after the 48 weeks of the trial. [17]
Duchenne muscular dystrophy is the most common type of muscular dystrophy, [3] with a median life expectancy of 27–31 years. [5] [11] However, with comprehensive care, some individuals may live into their 30s or 40s. [3] Duchenne muscular dystrophy is considerably rarer in females, occurring in approximately one in 50,000,000 live female ...
The trial is testing boys 2 to three years of age with DMD, a genetic muscle wa ... tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy(DMD), the drugmaker ...
Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. [3] It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells. [3]
(Reuters) -Sarepta Therapeutics said on Monday its new experimental drug for Duchenne muscular dystrophy (DMD) was far more effective in increasing levels of a key protein than its bestselling ...
Givinostat is in numerous phase II clinical trials (including for relapsed leukemias and myelomas), [15] and has been granted orphan drug designation in the European Union for the treatment of systemic juvenile idiopathic arthritis, [16] polycythaemia vera. [11] and Duchenne muscular dystrophy.
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