BioPharma Dive2d
Epicrispr banks $68M to test epigenetic editing on rare muscle disease
The startup is using CRISPR tools to stop errant expression of a gene linked to facioscapulohumeral muscular dystrophy, a disorder on the radars of several drugmakers.
GEN - Genetic Engineering and Biotechnology News1d
Epicrispr Biotechnologies to Begin Clinical Trials for its Lead Epigenetic Therapy for FSHD Patients
The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.
The American Journal of Managed Care9d
Exploring Care Needs, Physical Activity in Myotonic Dystrophy
Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...
Charity Today3d
Charities join forces on life-changing grants for children
The collaboration between Muscular Dystrophy UK, the leading charity for over 110,000 people in the UK living with one of ...
Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...