$48.7 billion for NIH, a 1% increase that helps sustain biomedical research critical to rare disease progress. The funding includes positive policy language limiting forward funding of grants and ...

Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today that the U.S. Food and Drug Administration (FDA ...

By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...

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 ...

TOKYO--(BUSINESS WIRE)--Duchenne Muscular Dystrophy (DMD) patients showed signs of disease progress slowing down, after oral consumption of Neu REFIX ß-glucan for 45 days along with routine ...

"Muscular dystrophy can take many things from me, but it will not take away my happiness, nor will it take away my faith." ...

Disability advocate TaLisha Gryzb is a mother of four living with muscular dystrophy who educates viewers about her ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...