Neuromuscular Disorders
Latest News
Course and outcome of Guillain-Barré syndrome measured in ongoing study
More than 1,500 patients have been enrolled so far.
Conference Coverage
Gene therapy for spinal muscular atrophy shows promise in early study
BOSTON – Early data show remarkable results for a gene therapy with few adverse events, in a phase I study.
Conference Coverage
Nusinersen for early spinal muscular atrophy: Final findings beat interim results
BOSTON – But the drug costs $750,000 a year, and questions remain about its use.
Conference Coverage
PATH study: Subcutaneous immunoglobulin safe, effective for CIDP maintenance
BOSTON – No differences in relapse recurrence were noted between low and high doses in the randomized, placebo-controlled, phase III study.
FDA/CDC
FDA approves first new drug for ALS in decades
Edaravone (Radicava) is only the second drug ever to be approved by the agency for amyotrophic lateral sclerosis.
Conference Coverage
VIDEO: About 1 in 20 ALS patients in Washington state chose assisted suicide
BOSTON – ALS patients were more likely to take deadly meds postprescription than were cancer patients.
Conference Coverage
VIDEO: Big research trials at AAN bring up important cost decisions
BOSTON – How will neurologists strike a balance between new drugs’ improved efficacy and safety and their affordability for patients?
Conference Coverage
AAN spotlights spinal muscular atrophy clinical research
A variety of sessions at this year’s annual meeting of the American Academy of Neurology in Boston will highlight efforts to treat children with...
From the Journals
Novel protein biomarker could accelerate ALS research
Researchers have shown evidence for the utility of measuring the mutated protein that is produced by a minority of ALS patients and tracking its...
Literature Review
Outpatient Visits Involving CNS Polypharmacy Rising Among Elderly
The number of outpatient visits involving CNS polypharmacy by adults aged 65 and older more than doubled between 2004 and 2013, especially among...
From the Journals
LVADs achieve cardiac palliation in muscular dystrophies
LVADs gain ground as destination therapy in muscular dystrophies.