Neuromuscular

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.

James Wymer, MD discusses his firsthand experience with risdiplam and identifies a key unmet need in the treatment of spinal muscular atrophy.

An expert neurologist emphasizes the importance of medication compliance and the value of multidisciplinary management in meeting patient goals of therapy for spinal muscular atrophy.

Arun S. Varadhachary, MD, PhD shares insights into clinicians’ and patients’ experiences with medical therapy, comments on unmet needs for adult patients, and speculates about the future of SMA care.

LX1004 and APB-102 are currently in the early phases of research to treat CLN2 Batten disease and SOD1 amyotrophic lateral sclerosis, respectively.

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Higher percentages of infants were event-free and were classified as having a motor-milestone response at month 12 than in historical cohorts.

Take 5 minutes to catch up on NeurologyLive's highlights from the week ending July 30, 2021.