Friedreich Ataxia Impacts Growth and Weight Gain in Adult and Pediatric Patients
In a prospective natural history study, researchers tested the association between body mass index and height and clinical characteristics in Friedreich ataxia.
Neuromuscular Disorders
Ravulizumab Gets Priority Review for Generalized Myasthenia Gravis
The application is supported by data from the phase 3 study that evaluated the efficacy and safety of ravulizumab-cwvz in 175 adults with generalized myasthenia gravis who were not previously treated with a complement inhibitor.
Vyvgart Approved for Generalized Myasthenia Gravis
Vyvgart is a neonatal Fc receptor antagonist designed to reduce IgG antibody levels and block the IgG recycling process.
FDA Approves First-in-Class Treatment for Myasthenia Gravis
Vyvgart (efgartigimod), a neonatal Fc receptor blocker, was approved to treat generalized myasthenia gravis in adults who test positive for the anti-acetylcholine receptor antibody.
EEG Identifies Clinical Subgroups Within Amyotrophic Lateral Sclerosis
Using similarity network fusion and spectral clustering, researchers sought to identify ALS disease subphenotypes and whether different patterns of disruption are predictive of outcome.
Can Recombinant Zoster Vaccine Cause Guillain-Barré Syndrome in Older Adults?
Using Medicare claims data, researchers determined whether there is an increased risk of developing Guillain-Barré syndrome following vaccination with the zoster vaccine.
Incidence, Prevalence, and Mortality of Dermatomyositis in Adults Determined
Using the ACR/EULAR criteria, researchers studied the population-based incidence, prevalence, and mortality of dermatomyositis in adults.
STR1VE-EU Trial Shows Efficacy of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
In a phase 3 trial, researchers assessed the safety and efficacy of onasemnogene abeparovec for symptomatic patients younger than 6 months with SMA1 and one or two SMN2 copies.
Patterns, Characteristics of Children Diagnosed With Acute Flaccid Myelitis Between 2014 and 2020
Researchers conducted a study that compared the patterns and characteristics of acute flaccid myelitis among patients diagnosed in peak years vs those diagnosed in nonpeak years.
Potential Safety Concern for Guillain-Barré Syndrome Identified for Ad26.COV2.S
Receipt of the Ad26.COV2.S COVID-19 vaccine is associated with a potential small, but statistically significant, safety concern for GBS.
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