Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.
Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
The plan put forth Tuesday could see the Sackler family pay up to $7 billion. Also in the news: Merck prepares to close its manufacturing plant in Pennsylvania; Optum Rx, which includes UnitedHealth ...
“Health Minute” brings original health care and health policy reporting from the KFF Health News newsroom to the airwaves each week. (3/18) Here's today's health policy haiku: ...
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...
Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...
Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...