Muscular Dystrophy News2d
FDA OKs trial to test gene therapy candidate in LGMD children
The FDA has given the green light to a clinical trial testing ATA-200, a gene therapy for limb-girdle muscular dystrophy ...
Muscular Dystrophy News1d
Being mindful of kidney stones while living with DMD
Columnist Betty Vertin hopes her family's experience with kidney stones might help you be better prepared while living with DMD.
Muscular Dystrophy News4d
Sarepta stops development of exon 51-skipping therapy for DMD
Despite positive trial data, Sarepta Therapeutics has decided to stop the clinical development of SRP-5051 (vesleteplirsen), ...
Muscular Dystrophy News10d
Supporting artists with disabilities through a new business venture
Columnist Shalom Lim shares how his business, Rebirth Ensemble, will allow him to pursue visual arts and support artists with ...
Muscular Dystrophy News13d
I always try to pause to appreciate the small victories
Columnist Robin Stemple says he chooses to focus on what FSHD progression hasn't taken from him. The small victories matter, ...
Muscular Dystrophy News8d
Patients with certain mutations lose their walking ability earlier: Study
Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...
Muscular Dystrophy News8d
The missed milestones in the teenage years because of DMD
Columnist Betty Vertin wishes she could take away the pain over the milestones in the teenage years her sons with DMD will miss.