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 ...
Mayo Clinic's Translational Neuromuscular Disease Research Lab led by Nathan P. Staff, M.D., Ph.D., studies the biological ...
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 ...
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 ...
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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