Neuromuscular

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 27, 2021.

Investigators found incobotulinumtoxinA treatment decreased unstimulated salivary flow rate and increased GICS ratings in children.

The chief medical officer of Wave Life Sciences outlined the FOCUS-C9 study, which has incorporated feedback from patients with both ALS and FTD, as well as investigators in the research field.

The long-acting C5 complement inhibitor, developed by Alexion and approved for atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria (PNH), failed to show an effect on ALSFRS-R score.

The potential of a new investigational treatment for C9orf72-associated amyotrophic lateral sclerosis and frontotemporal could offer long-lasting effects for patients.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 20, 2021.

The chief medical officer of Wave Life Sciences spoke on the FOCUS-C9 study and the potential of WVE-004 to generate positive outcomes for patients.

The potential of a new investigational treatment, WVE-004, was discussed by the chief medical officer of Wave Life Sciences, the company that developed the molecule for C9-ALS and C9-FTD.

Program chairs Fred D. Lublin, MD, and Stephen Silberstein, MD, offer insight into the third annual IFN meeting, which is set for September 17-18, 2021, to be held in a virtual setting.

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Neurology News Network for the week ending August 14, 2021.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 13, 2021.

The chief medical officer of Wave Life Sciences, Michael Panzara, MD, MPH, discussed the potential of an investigational new treatment, WVE-004, for patients with C9orf72-associated ALS and FTD.

Michael Panzara, MD, MPH, chief medical officer of Wave Life Sciences, spoke on the FOCUS-C9 study, which recently began dosing of an investigational treatment, WVE-004, for patients with ALS and FTD.

The autologous, expanded Treg cell therapy was found safe and tolerable in a phase 1 study of 3 patients with ALS.

Following the commencement of a study exploring WVE-004, an investigational treatment for C9-ALS and C9-FTD, the director of the Sean M. Healey & AMG Center for ALS discussed the clinical implications and the treatment’s potential.

Two unblinded interim analyses by the Data Monitoring Committee are planned to assess the futility of reldesemtiv, with the first occurring 12 weeks after one-third of the sample size is randomized.

The director of the Sean M. Healey & AMG Center for ALS outlined the trial, which will evaluate ideal dose level and frequency of the investigational treatment for patients with C9-ALS and C9-FTD.

Investigators obtained a molecular diagnostic yield of 39% in 66 sequential, unselected individuals with diverse presentations of rare neurogenetic disorders when using a model that integrates additional assays.

In addition to the uplifted phase 1 study, Novartis also initiated the phase 3 STEER study, which will evaluate OAV-101 in treatment-naive patients with SMA type 2 aged between 2 and 18 years old.

Developed by Wave Life Sciences, the trial of the investigational treatment, WVE-004, seeks to identify ideal dose level and frequency in patients with C9orf72-associated amyotrophic lateral sclerosis and FTD.

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Take 5 minutes to catch up on NeurologyLive's highlights from the week ending August 6, 2021.

Branded as Nexviazyme, Sanofi’s enzyme replacement therapy demonstrated key improvements in measures of disease burden while maintaining its known safety profile.