The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...

A quarter of a million Americans are living with muscular dystrophy or a related neuromuscular disorder. It's the reason ...

Nationwide Children's Hospital has renamed its gene therapy research program and clinics for Dr. Jerry Mendell, who devoted ...

Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

Dr. Laila Bastaki, Head of the Genetic Diseases Center at the Ministry of Health, emphasized the importance of incorporating ...

Forge Biologics will help with the manufacturing of adeno-associated viruses (AAVs) in a collaboration with the non-profit ...

BofA Securities has upgraded Fulcrum Therapeutics (NASDAQ:FULC) ahead of the company’s phase 3 losmapimod readout, expected ...

Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect ...

The biotech will now suspend development of a drug prospect that Sanofi acquired partial rights to just four months ago.

Meet Sehba Shah, Pakistan's inspiring woman chess player who lives with muscular dystrophy yet dreams high above the ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...