Neuromuscular

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.

Here's what is coming soon to NeurologyLive.

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.

Here's what is coming soon to NeurologyLive.

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.

Neurology News Network for the week ending June 5, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending June 4, 2021.

Tom Crawford, MD, examines data from the SUNFISH trial of patients with type 2 and type 3 SMA and emphasizes the need for further data before risdiplam can be directly compared to other treatment options.

Basil Darras, MD discusses the FIREFISH trial of risdiplam in infants with type 1 symptomatic SMA and comments on the difficulty of comparing agents when trials have had different enrollment criteria.

Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital discussed how the PHOENIX study expands upon the previous study, CENTAUR.