While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Muscular dystrophy (MD) represents a group of inherited muscle disorders that primarily cause muscle weakness and muscle ... Some types of muscular dystrophy can be fatal. Certain types, including ...

This status is awarded by the… Capricor Therapeutics has completed its submission of a biologics license application (BLA) seeking U.S. approval of deramiocel, the company’s cell therapy for heart ...

On July 18, 2025, The Speak Foundation will host the fourth International Limb Girdle Muscular Dystrophy (LGMD) Conference at the J.W. Marriott Orlando, Grande Lakes. The International LGMD conference ...

Initiation of Phase 1b study using SGT-212 for the treatment of patients with Friedreich's Ataxia expected in 2H25. Click ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

Quince Therapeutics, Inc. , a late-stage biotechnology company dedicated to unlocking the power of a patient's own biology for the treatment of rare diseases, announced the online publication of ...

The methods and protocols generated in this study are accessible and can be implemented in any standard research laboratory without the need for ...

Discover the latest addition to Children's Hospital Los Angeles' 10 cell and gene therapy treatments, including Casgevy for sickle cell disease ...

NDAQ:SLDB) Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich's Ataxia ...

Jan. 21, 2025 — Trypanosomes are parasites that cause sleeping sickness, Chagas disease, and various animal diseases. Diagnosis and treatment remain complex and no effective vaccine has been ...