Neuromuscular

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.

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.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 28, 2021.

Six months after being discharged from the hospital for COVID-19, patients scored worse than average on 1 or more NeuroQoL scale components, which measured anxiety, sleep, fatigue, and depression.

The long-term follow up of the phase 1 START trial found that previously achieved motor milestones were preserved over a 5-year period.

Matthew B. Harms, MD, associate professor of neurology at Columbia and medical consultant for the Muscular Dystrophy Association, shares his thoughts on the current state of ALS care.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending May 21, 2021.

"Mind Moments," a podcast from NeurologyLive, brings you an exclusive interview with Matthew B. Harms, MD.

The trial design was presented at the 2021 ENCALS meeting and is set to run for 48 weeks at more than 50 clinical centers.

The investigational drug, previously granted fast-track designation, did not meet primary or secondary end points in the ORARIALAS-01 trial.

Copy number results for the kit were highly concordant with reference results from other methods.

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The 2 organizations will use whole-genome sequencing and clinical characterization to investigate the genetic causes of CP.