Patients who received the drug combination “retained higher levels of physical function over 6 months compared with those who received placebo.”...
The approval marks the second drug therapy for Duchenne muscular dystrophy.
This allows for more consistent therapy for children with cerebral palsy who have upper- and lower-limb spasticity.
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...
Early signs of neurodegeneration despite normal brain function detected in young adult carriers of the Huntington’s disease gene.
From the Journals
In a cohort of patients with dermatomyositis, 8% did not have a rash at the time of muscle biopsy.
A case report from China suggests a possible association between GBS and SARS-CoV-2 infection.
“What happens in [the] vagus doesn’t just stay in vagus, but will actually affect our emotions in different ways,” Dr. Cryan said.
In patients with Parkinson’s disease, dementia, and neuropathy, higher out-of-pocket drug prices were associated with lower adherence for common...
News from the FDA/CDC
The agency also authorized the first newborn screening test for DMD.
Patients who undergo the procedure appear not to have progressive or delayed complications during long-term follow-up.