Neuromuscular Disorders
Conference Coverage
Synthetic triglyceride shows potential in Huntington’s disease
An extension study found that the synthetic medium-chain triglyceride triheptanoin improved brain energy and caudate atrophy in Huntington’s...
Literature Review
Potential first-in-class, targeted therapy for myasthenia gravis
Results from the phase 3, randomized, placebo-controlled ADAPT trial showed reductions in disease burden and improvements in strength and quality...
Literature Review
Basal ganglia microcircuits offer clues to Parkinson’s symptoms
In a mouse model of Parkinson’s disease, specific neuronal subpopulations, or microcircuits, in the basal ganglia could be manipulated to...
From the Journals
Neurologic disorders ubiquitous and rising in the U.S.
Most of the rise in disease burden could be attributed to population aging, while age-standardized figures showed stroke and dementias to be level...
News from the FDA/CDC
FDA approves first targeted treatment for rare DMD mutation
This particular mutation of the DMD gene “is amenable to exon 45 skipping.”
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.