Entrada has secured the authorisation within the EU Clinical Trial Regulation to begin the Phase I/II trial of ENTR-601-45.

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new DMD gene therapy.

Autolus' CAR-T cell therapy for a rare and aggressive form of acute lymphoblastic leukaemia (ALL), Aucatzyl, has been ...

The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy ... specifically target genetic mutations like Sarepta Therapeutics ...

DMD treatment SAT-3247 was safe and well-tolerated, and showed signs of increasing muscle strength in five adults, Satellos ...

A rare and fatal form of muscular dystrophy has long posed a devastating diagnosis for children, primarily affecting boys.

Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Genetic testing for hereditary cancer unexpectedly uncovered a case of myelodysplastic syndrome (MDS), highlighting the need ...

A 46-year-old woman with type 1 myotonic dystrophy (DM1) presented to the emergency department with dyspnea and palpitations.

The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.