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 ...
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 ...
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 ...
EVRY, France--(BUSINESS WIRE)--Atamyo Therapeutics a clinical-stage biotechnology company focused on the development of new generation gene therapies targeting muscular dystrophies and ...
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 ...
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 ...
Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...
PALO ALTO, Calif., June 26, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced ...
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Some children to access new muscular dystrophy drug
Some children in Northern Ireland who live with a severe form of muscular dystrophy will be able to access new medication on the health service, BBC News understands. The move from the Belfast Health ...
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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