Neuromuscular Disorders
Literature Review
New steroid dosing regimen for myasthenia gravis
Rapid tapering of prednisone is recommended in patients requiring combined corticosteroid and azathioprine therapy.
Guidelines
Joint guidelines favor antibody testing for certain Lyme disease manifestations
The Infectious Disease Society of America, American Academy of Neurology, and the American College of Rheumatology teamed up for the first time to...
Conference Coverage
Gene-replacement therapy shows promise in X-linked myotubular myopathy
An investigational gene therapy decreases ventilator dependence and may improve motor development in children with a rare, life-threatening...
Conference Coverage
Ataluren delays disease milestones in patients with nonsense mutation DMD
Patients who received ataluren in addition to standard of care were 2.2 years older when they lost ambulation, compared with patients receiving...
Conference Coverage
Acute flaccid myelitis: More likely missed than diagnosed
A retrospective series reports that 78% of children with acute flaccid myelitis are misdiagnosed initially, causing delays in treatment.
Conference Coverage
Nusinersen provides continued benefits to presymptomatic children with SMA
Most children developed normally in an interim analysis of the NURTURE trial that did not find any new safety concerns.
Literature Review
Drug combo slows functional decline in ALS
Patients who received the drug combination “retained higher levels of physical function over 6 months compared with those who received placebo.”...
FDA/CDC
FDA approves viltolarsen (Viltepso) for Duchenne muscular dystrophy
The approval marks the second drug therapy for Duchenne muscular dystrophy.
News
FDA expands Dysport use for cerebral palsy–related spasticity
This allows for more consistent therapy for children with cerebral palsy who have upper- and lower-limb spasticity.
Literature Review
First reported U.S. case of COVID-19 linked to Guillain-Barré syndrome
The unique clinical features in the U.S. case are urinary retention secondary to dysautonomia and ocular symptoms of...
Literature Review
Huntington’s disease biomarkers appear 24 years before clinical symptoms
Early signs of neurodegeneration despite normal brain function detected in young adult carriers of the Huntington’s disease gene.