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

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

A mother's love drives her to pursue experimental treatment as two of her four children battle a rare genetic disease that ...

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

Dr. Mackey’s project, “ Regenerating healthy human skeletal muscle at single nucleus resolution ,” will create the most detailed molecular map to date of how healthy human muscle repairs itself. The ...

In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...

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 ...

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

Quadriplegic and bedridden in a prefabricated home, 36-year-old Li Xia can only move one finger and one toe -- yet he runs a ...

Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a progressive muscle‑wasting disorder showed sustained benefit in ...

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 ...