Charity Today3 天
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 ...
Stirling school 'goes orange' to pay tribute to pupil battling rare condition
The 11-year-old's strength and bravery in living with the impact of the serious muscle-wasting condition was hailed by her ...
BioPharma Dive2 天
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 News1 天
Epicrispr Biotechnologies to Begin Clinical Trials for its Lead Epigenetic Therapy for FSHD ...
The therapy, EPI-321, is intended to treat cases of facioscapulohumeral muscular dystrophy, a genetic neuromuscular disorder.
Brave Stirling schoolboy with rare condition gets special send off ahead of move to high school
Convener of Stirling Council’s Children and Young People Committee, Cllr Danny Gibson said: “Somhairle’s bravery, inner strength and positivity in the face of this serious condition has had a major ...
'I'm one of first in world to try new therapy for incurable muscle disorder FSHD - and I ...
When Lauren Clarke was 14, she was diagnosed with a rare, incurable muscle-weakening disorder. Ten years on, she has become ...
The American Journal of Managed Care10 天
How Access to SMA Treatment Varies Globally and by Insurance Type
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...
The American Journal of Managed Care10 天
Exploring Care Needs, Physical Activity in Myotonic Dystrophy
Patients with myotonic dystrophy face unique challenges in managing their ... 1-2 Recent studies presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference shed ...