Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...

Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy.

Detailed price information for Denali Therapeutics Inc (DNLI-Q) from The Globe and Mail including charting and trades.

Amicus Therapeutics (FOLD) remains a Buy, driven by strong product momentum and new assets. Read here for more investment ...

Dr. Drucy Borowitz helped turn cystic fibrosis from a deadly childhood illness into a treatable condition, giving families ...

HYDERABAD, India, Oct. 7, 2025 /PRNewswire/ -- The Rare Disease Therapeutics Market is experiencing an unprecedented wave of innovation, driven by gene therapies, RNA-based treatments, enzyme ...

The Batten Disease pipeline presents opportunities with over 10 companies developing 12+ drugs in varied clinical stages. Novel approaches, including gene therapy and enzyme replacement, address unmet ...

BioMarin Pharmaceutical BMRN reported third-quarter 2025 adjusted earnings per share (EPS) of 12 cents against the Zacks ...

Australians with the extremely rare and life-threatening inherited disease, acid sphingomyelinase deficiency (ASMD) will now have free access to a ...