Shalom Lim is a University of Liverpool graduate, having completed an honors bachelor’s degree in criminology and security in July 2021. Born with Duchenne muscular dystrophy and diagnosed at 4 months ...
Arrakis Therapeutics’ investigational RNA-targeted small molecule (rSM) therapies were found to work as intended in preclinical studies and eased symptoms in a mouse model of myotonic dystrophy type 1 ...
Agamree (vamorolone) has been recommended for use in the National Health Service (NHS) in England, Wales, and Northern Ireland for treating Duchenne muscular dystrophy (DMD) patients, 4 and older. “We ...
Living with Duchenne muscular dystrophy (DMD) has involved a series of challenges and triumphs, each shaping the person I am today. As 2025 approaches, I’m reflecting on that journey as well as ...
The investigational oral therapy sevasemten significantly reduced markers of muscle damage in people with Becker muscular dystrophy (BMD) treated in a Phase 2 trial, according to top-line results ...