Seven-year-old Archie Ennis has touched the hearts of the nation as his family plea for funds to help him get the life-saving ...

Having found that in biomedical research, the Bird Rock resident and scientist at La Jolla’s Sanford Burnham Prebys has ...

Most individuals diagnosed with a rare disease won’t live to become teenagers or adults. I knew what I had to do.

The Minnesota Department of Health has officially added two more diseases to the list of conditions for which newborns are ...

Dyne Therapeutics’ DYNE-101 shows positive results in phase 1/2 ACHIEVE study. Read why DYN stock may rise with FDA approval and a $2.78B market opportunity.

Dominic Olsen is a bright and determined 10-year-old boy living with Duchenne Muscular Dystrophy, a rare disease that has ...

Duchenne muscular dystrophy (DMD) is a genetic condition that primarily affects people assigned male at birth. However, due to the condition’s X-linked inheritance pattern, only people assigned ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and ...

Look ahead to this year's Muscular Dystrophy Association (MDA) meeting, which will feature discussions on the latest gene therapies, clinical trial data, policy considerations, and more in the realm ...

NEW YORK, NY / ACCESS Newswire / March 13, 2025 / Levi & Korsinsky notifies investors that it has commenced an investigation of PepGen Inc.

Regenxbio Inc (RGNX) showcases robust financial positioning and strategic progress in gene therapy, despite challenges in ...

The skeletal phenotype in Becker muscular dystrophy: The under-studied cousin of Duchenne Title: P19: Myosin inhibitor EDG-4131 improves pathophysiology and molecular pathology in BMD model mice ...