ET HealthWorld14h
Parents of muscular dystrophic patients protest in Delhi, demand access to high-cost treatment
One of the demands raised during the protests was access to 'Elevidys', a one-time gene therapy treatment that has the ...
A mother’s battle: Advocating for life-saving treatments for children with Duchenne muscular dystrophy
When her son reached three last year, a new name loomed over their lives — Duchenne Muscular Dystrophy (DMD), a ...
pharmaphorum4h
Multiple studies of Sarepta's DMD therapy halted after death
The EMA has called a temporary halt to a phase 2 trial of Sarepta's Roche-partnered Duchenne muscular dystrophy (DMD) gene ...
Duchenne muscular dystrophy's impact on the brain may be reversible
New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...
Managed Healthcare Executive2d
Wave’s Duchenne Therapy Has Promising Phase 2 Results
Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.
The Pharma Letter6h
EMA halts Sarepta’s DMD gene therapy trial after patient death
The European Medicines Agency has temporarily suspended enrollment and dosing in a Phase II trial of Sarepta Therapeutics' ...
STAT2h
Pharmaceuticals are spared from tariffs, for now
President Trump yesterday announced a baseline tariff of 10% on all imported products and higher rates on certain countries ...
Edgewise Therapeutics: Despite AF Safety Issues, EDG-7500 Advancement Presses On
Discover growth opportunities with Edgewise Therapeutics, Inc. in the hypertrophic cardiomyopathy market. Click for my EWTX ...
Fierce Pharma1h
After patient death, Sarepta and Roche pause 3 trials of gene therapy Elevidys in Europe
In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...
Managed Healthcare Executive1d
New Study Shows Duchenne's Effect on the Brain Might Be Reversed
Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...
Defeat Duchenne Canada Launches 2025 Research Grant Cycle, Set to Commit its $20 Millionth Dollar to Groundbreaking Research
Defeat Duchenne Canada, the country's leading funder of Duchenne muscular dystrophy (DMD) research, is set to begin accepting innovative research proposals as of March 31, 2025. They are seeking ...