Russian FSHD registry shows moderate inverse correlation between D4Z4 repeats and clinical severity
What does FSHD look like in Russia? A registry study maps the disease
An observational cohort study of 470 patients in the Russian FSHD Patient Registry found genetic confirmation in 76% of participants.
Nearly half of Russian FSHD patients first notice shoulder blade weakness, while others start with facial symptoms or a classic childhood fo…
Tamoxifen adjunct to corticosteroids shows no motor benefit in Duchenne muscular dystrophy extension study
Does adding tamoxifen to standard treatment help boys with Duchenne muscular dystrophy?
A 48-week open-label extension of a phase 3 trial in 66 boys with DMD found no significant difference in motor function change between early…
Adding tamoxifen to standard steroid treatment did not improve motor function or slow disease progression in boys with Duchenne muscular dys…
Phase 3 RCT: Ataluren shows favorable trend for slowing functional decline in nonsense mutation DMD
Can a drug help boys with Duchenne muscular dystrophy walk longer?
A phase 3 RCT of 359 people with nonsense mutation Duchenne muscular dystrophy found that 72 weeks of ataluren treatment was associated with…
Boys with a specific genetic form of Duchenne muscular dystrophy who took ataluren saw their walking ability decline less over 72 weeks than…
Phase 2 trial of oral ifetroban in DMD cardiomyopathy reports no safety results yet
Can a new pill help protect the hearts of boys with Duchenne muscular dystrophy?
A phase 2 randomized, double-blind, placebo-controlled trial evaluated low- and high-dose oral ifetroban in 46 males with DMD-associated car…
A new pill called ifetroban is being tested to safely treat heart weakness in boys with Duchenne muscular dystrophy, addressing a leading ca…