Please provide your email address to receive an email when new articles are posted on . Ataluren preserved hand-to-mouth function in boys with nonsense mutation Duchenne muscular dystrophy. Ataluren ...

Please provide your email address to receive an email when new articles are posted on . RGX-202 was safe and well-tolerated in all 12 patients at two different doses with no serious adverse events.

Genetic testing, often via a blood sample, can confirm a muscular dystrophy diagnosis. It can also identify specific gene mutations to help guide treatment and determine whether or not someone is a ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Avidity Biosciences’ AOC 1020, newly dubbed del-brax, demonstrated a safe profile and was tied to functional improvement among people with facioscapulohumeral muscular dystrophy (FSHD) in a phase 1/2 ...

—Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients with Duchenne muscular dystrophy using non-invasive ventilation. Individuals ...

Doctors use physical therapy to treat Duchenne muscular dystrophy. It can help maintain muscle function, boost quality of life, and prevent complications. A physical therapist will tailor your ...

CAMBRIDGE, Mass., Sept. 07, 2023 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. ® (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically ...

-- Continued functional improvements wereobserved at 18 months in the low-dose cohort-- -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration -- -- ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...