REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast on Thursday, May 14, 2026, at 8:00 a.m. ET to discuss topline results from the pivotal trial of RGX-202, the company's ...

Managing the transition to mobility aids is a pivotal part of life with DMD. Discover expert strategies for maintaining ...

PHOENIX - We are learning more about the death of comedian Gilbert Gottfried, which was announced on April 12. According to a statement from Gottfried's longtime friend and publicist, Glenn Schwartz, ...

Investigational deramiocel is back under FDA review again ...

Muscular Dystrophy Association Marks ALS Awareness Month throughout May with Breakthrough Research, Powerful Stories, and ...

Best disease is an eye condition that people inherit from their parents. It starts during childhood and causes progressive vision loss later in life, developing in six stages. Best disease, also known ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

Muscular dystrophies represent a group of inherited primary diseases of muscle, characterized by muscle fiber degeneration and muscle weakness. Classification of these conditions has traditionally ...

For Duchenne and Becker muscular dystrophies, muscle biopsy may show whether dystrophin, a muscle protein, is missing or abnormal, and DNA testing is used to analyze the condition of the related gene.

Tributes poured in for Gilbert Gottfried on Tuesday after news broke that he died in Manhattan. The 67-year-old comedian’s family shared on Twitter that he passed away “after a long illness.” ...