Neuromuscular

The exploratory end point of the ability to thrive in symptomatic patients was met by 30% of those in a subanalysis of STR1VE-EU, compared to none in the comparative Preclinical Neuromuscular Clinical Research study.

Julie Parsons, MD, comments on the role of primary care providers and gene testing for the early diagnosis of spinal muscular atrophy.

Identifying spinal muscular atrophy through early diagnosis using prenatal and newborn screenings is discussed.

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The Distinguished Professor of Neuromuscular Disease at UNC School of Medicine spoke to the results of the phase 3 ADAPT trial, and what else he’s like to see investigated with efgartigimod.

Neurology News Network for the week ending June 19, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending June 18, 2021.

The Distinguished Professor of Neuromuscular Disease at UNC School of Medicine discussed the findings of the ADAPT trial and the promise efgartigimod has shown to be an effective agent for generalized myasthenia gravis.

Santhera and ReveraGen announced findings of the phase 2b VISION DMD study of the investigational therapy.

ALS has only recently been associated with causative or disease-modifying mutations in 20 genes that encode proteins with diverse functions, with promising therapeutic targets including proteins in pathways that regulate protein homeostasis.

The nurse practitioner at the Woodruff School of Nursing at Emory University detailed why a cultural shift is needed to incorporate APCs into the neurology space and thus improve multidisciplinary care.

Julie Parsons, MD, discusses the importance of a multidisciplinary team approach to managing SMA in clinical practice.

John Brandsema, MD, leads the discussion on the 5 main types of spinal muscular atrophy and the evolution of disease classification.

The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital also discussed further research that needs to be done in ALS.

A pharmacokinetic/pharmacodynamic model indicated that the exposures predicted with a higher dose of nusinersen may lead to more clinically meaningful increases of at least 5 points on CHOP INTEND score.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending June 11, 2021.

A panel of experts considers current unmet needs in SMA by emphasizing the need for further study of combination therapy, more comprehensive newborn screening, and more effective early diagnosis and treatment.

Key opinion leader in neurology Claudia Chiriboga, MD, MPH provides insight into response to risdiplam in patients with SMA who were not naïve to treatment as studied in the JEWELFISH clinical trial.

The nurse practitioner at the Woodruff School of Nursing at Emory University detailed why advanced practice clinicians should be utilized more in neurology settings.

The HINE-2 motor milestone total score in those treated with nusinersen increased steadily from a baseline mean of 1.46 to 11.86 at day 1135, representing a clinically significant change of 10.43 points.

Final thoughts from leading experts on the future of treating Duchenne muscular dystrophy and keeping standard of care in the forefront.

Recent approval of casimersen, its administration, efficacy, safety, and patient selection for treatment of Duchenne muscular dystrophy.

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The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital discussed how adding AMX0035 to a therapeutic regimen might improve treatment of ALS.