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 ...
EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and ...
Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9) Designation supports development of CRD-003, an ...
University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular dystrophy. Published in the journal Nature Communications, the study provides ...
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 ...
Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...
If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When ...
WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the ...
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