Researchers to Study Optimized Home-Based Respiratory Management in ALS
Researchers from South America presented a protocol for a randomized trial to study the effects of an optimized home-based respiratory care approach in patients with ALS.
Neuromuscular Disorders
SARS-CoV-2 May Be Linked to Immune-Mediated Myopathy
In this case-control autopsy series, study researchers evaluated skeletal muscle and myocardial inflammations in patients with severe COVID-19 who died.
Exservan Now Available for Amyotrophic Lateral Sclerosis
According to the CDC, more than a thousand cases of myocarditis and pericarditis following mRNA COVID-19 vaccination have been reported to the Vaccine Adverse Event Reporting System since April 2021.
Long-term Safety of Onasemnogene Abeparvovec in Spinal Muscular Atrophy
Study researchers sought to assess the safety of onasemnogene abeparvovec for the treatment of infants with spinal muscular atrophy.
New Gene Variants Identified That May Raise Risk for ALS
Variants in TP73 are associated with risk for amyotrophic lateral sclerosis (ALS), according to a study published online.
Apitegromab Fast Tracked for Spinal Muscular Atrophy
Apitegromab is a selective inhibitor of the inactive form of myostatin to block its activation in the muscle.
Accelerated Neuromuscular Aging: Effect of Type 1 Diabetes on Muscle Function and Morphology
A team of investigators from McMaster University assessed the effect of aging on skeletal muscle function in individuals with and without type 1 diabetes.
AChR Ab Levels Predict 1-Year Immunosuppressive Therapy Success in Myasthenia Gravis
A team of investigators evaluated the relationship between changes in AChR Ab levels induced by immunosuppressive treatment and myasthenia gravis prognosis at 1-year following treatment in patients with the disease.
Efgartigimod Well Tolerated and Effective for Myasthenia Gravis
Efgartigimod may be an effective and well-tolerated treatment option for patients with myasthenia gravis.
Gene Therapy Makes Strides for Infants With Spinal Muscular Atrophy
During the AAN 2021 Virtual Annual Meeting, investigators presented their findings on the safety and efficacy of onasemnogene abeparvovec (formerly AVXS-101) in presymptomatic SMA patients with 3 copies of the survival motor neuron 2 gene.
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