Decker was given the 2025 MDA Legacy Award, recognizing his efforts to advance gene therapy, scientific research, and the ...

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

Activating a protein called potassium channel Kv2.1 may help improve motor function in SMA, making it a potential SMA ...

The mother of a teenage boy, with muscular dystrophy, said her son hasn’t been able to leave their Oakland home for almost a ...

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

July 17, 2024 — A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic ...

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

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with an intrathecal formulation intended for older patients.

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 ...

WEDNESDAY, March 19, 2025 (HealthDay News) -- A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment. The ...