"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...

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 ...

-- Continued functional improvements wereobserved at 18 months in the low-dose cohort-- -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration -- -- ...

Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.

Dyne Therapeutics started the new year with proof-of-concept clinical data for experimental genetic medicines addressing two rare muscle diseases—one of which has no FDA-approved treatments. Now the ...

A person may inherit the genetic changes responsible for muscular dystrophy. These genetic changes can also occur due to spontaneous genetic mutations. In either case, the disease is not preventable.

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 ...

CORAL GABLES, Fla. — There’s new hope for children born with a fatal form of muscular dystrophy. Nicklaus Children’s Hospital is one of the first in the nation and one of only three hospitals in ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting muscular dystrophies and cardiomyopathies, today ...

Oculopharyngeal muscular dystrophy (OPMD) is a rare inherited disorder that causes weakness in the eye and throat muscles. It may lead to drooping eyelids and swallowing difficulties. Muscular ...

EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and ...

FDA and Capricor aligned on endpoints for HOPE-3 pivotal trial HOPE-3 pivotal trial completed; topline data expected mid-Q4 2025 to support BLA resubmission Company preparing to resubmit CRL response ...