By John Lauritsen Click here for updates on this story Minnesota (WCCO) — An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Preclinical data with the investigational gene therapy INS1202 show improvements in survival and motor performance in ALS.

The Family Achievement Center will offer family-oriented physical, occupational and speech therapies once it opens in May.

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study, which is examining ...

The company behind the drug, Sarepta Therapeutics, said that the patient suffered acute liver injury, a known side effect, ...

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) ("Satellos" or the "Company"), a biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and ...

UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living ...

UCB, a global biopharmaceutical company, today announced positive data from studies involving its investigational pyrimidine nucleoside therapy, doxecitine (dC) and doxribtimine (dT), in people living ...

Roche to present latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association 2025 conference: Basel Tuesday, March 18, 2025, 11:00 Hrs [IST] ...

This weekend kicked off the MDA annual Clinical & Scientific Conference, bringing together the neuromuscular disease ...