Capricor Therapeutics faced a significant setback on July 11, 2025, when the FDA rejected its cell therapy, Deramiocel , for treating...

The FDA has declined to approve Ultragenyx Pharmaceutical's gene therapy, UX111, for treating Sanfilippo syndrome type A, a rare genetic...

Biogen’s experimental drug, Salanersen, is showing promising potential for treating children with spinal muscular atrophy (SMA), even...