People frequently wait years to be "taken seriously," postcode lotteries for access to care and a general lack of knowledge are among some of the key findings from a University of Aberdeen ...
The degree to which novel value elements such as insurance value impact estimated treatment value for rare, severe genetic diseases such as Duchenne muscular dystrophy is unclear. Objectives: To ...
Capricor Therapeutics announced that the U.S. FDA has granted Orphan Drug Designation to its lead cell therapy candidate, Deramiocel, for the treatment of Becker Muscular Dystrophy (BMD). This ...
New research shows how muscular dystrophy (DM1) damages the heart over time, stressing the need for early diagnosis and ...
The Indiana-based Muscular Dystrophy Family Foundation (MDFF) is now accepting applications for its annual educational scholarship, which supports Indiana residents living with muscular dystrophy and ...
A 15-year-old boy in New Market with a neuromuscular disorder received a wheelchair accessible van last week through the support of family, community and a nonprofit dedicated to assisting those with ...
The U.S. Food and Drug Administration has approved the first generic version of Emflaza (deflazacort) oral suspension for Duchenne muscular dystrophy (DMD). Approval of the generic version of Emflaza ...
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