The MarketWatch News Department was not involved in the creation of this content. PARAMUS, N.J., March 9, 2026 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma, New Jersey, USA; President, Yukiteru Sugiyama ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

The investigational gene therapy SGT-003 is showing early signs of safety and efficacy for Duchenne muscular dystrophy in a ...

This time, her baby did test positive for SMA type 1, a severe neurodegenerative genetic condition that typically results in ...

Regenxbio Inc (RGNX) showcases robust pipeline progress and financial stability, despite regulatory challenges.

On World Rare Disease Day, we take a look at Pompe disease - a rare genetic disorder caused by a deficiency of the GAA enzyme, leading to glycogen buildup in muscles. It results in progressive muscle ...

The president and CEO of the Muscular Dystrophy Association talks about what she's most excited about in neuromuscular ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

The article explains why early dental care supports oral health, preventive care, and healthy development for a child's teeth.

Positive data from EXPERTS-ALS could accelerate RT1999 towards a registration trial; RT1999 to be presented as a poster ...

Keros Therapeutics will use the Massachusetts General Hospital MyMatch program to design a clinical trial testing rinvatercept for ALS.

ELMIRA, N.Y. (WETM)— Last week, Eric Dane passed away from ALS, also known as Lou Gehrig’s disease. He publicly shared his ...