A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...

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

R1 calpain3-related is extremely rare, and its prenatal management is unclear. A primigravida in her late 30s with LGMD R1 calpain3-related was referred to our hospital during the first trimester.

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials set to ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...