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

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 ...

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

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

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

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 ...

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.

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

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

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

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 ...