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 ...
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 ...
Last Saturday, I performed a monologue titled “Connection” at the Enabling Lives Festival here in Singapore. ART:DIS, a local nonprofit that uses the arts to support people with disabilities, ...