A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

ALBANY — University at Albany scientists have been awarded a $2.5 million grant from the National Institutes of Health to advance research to cure myotonic dystrophy — the most common form of ...

NEW YORK and SAN DIEGO, June 12, 2019 /PRNewswire/ -- The Muscular Dystrophy Association (MDA) and Locana, a leading RNA-targeting gene therapy company, today announced the award of an MDA Venture ...

ALBANY, N.Y. (NEWS10) — Scientists at the University at Albany (UAlbany) have been awarded a $2.5 million grant from the National Institutes of Health to advance research to cure myotonic dystrophy ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...

Myotonic Dystrophy Type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely affects multiple organs including skeletal muscle, heart, brain and the ...

PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and ...

Astellas has been haunted by patient deaths in a previous trial for a tricky neuromuscular condition, but that hasn’t stopped the Japanese Big Pharma from seeking out another gene therapy to target ...

In muscular dystrophy type 1, mutated DNA begets toxic RNA that alter liver function, including susceptibility to fatty liver disease and hypersensitivity to drugs. Understanding these effects is ...