Researchers at the Experimental and Clinical Research Center in Berlin are developing a targeted treatment for muscular ...
The results suggest that prolonging ambulation may not adversely impact cardiac function in adulthood for patients with ...
ECRC researchers developed a CRISPR-based gene therapy targeting dysferlin mutations in muscular dystrophy. By editing and ...
Duchenne muscular dystrophy is a serious condition that causes progressive muscle weakness. It affects 2,500 boys and men in ...
MDA continues to empower the next generation of leaders by supporting higher education opportunities for students living with ...
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host its sixth in-person Duchenne Healthcare Professionals ...
The Scottish Medicines Consortium (SMC) approved the drugs for use by NHS Scotland.
The biotech had been testing sevasemten, an orally administered fast skeletal myosin inhibitor, in a trial of 40 adults and 29 adolescents with Becker muscular dystrophy. Edgewise described the ...
Altruism values for treatments of rare, severe pediatric diseases have not been estimated. This study found the altruism value for a hypothetical new Duchenne muscular dystrophy treatment to be $80 ...
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