Neuromuscular

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.

An insight into the goals of therapy and treatment selection between oral and intrathecal medications for adult-onset spinal muscular atrophy.

The label now includes the use of ultrasound as a muscle localization technique in adults with spasticity, and includes muscles of the elbow, forearm, hand, and thumb.

A discussion of the multidisciplinary treatment approach in spinal muscular atrophy.

A specialist in the care of patients with spinal muscular atrophy reviews the CHERISH trial of nusinersen and examines the drug’s safety and efficacy profiles.

A key opinion leader in neurology highlights the value of a multidisciplinary approach to care for patients with spinal muscular atrophy in order to maximize a patient’s function through physical and medical therapy.

Patients with neurological sequelae from COVID-19 were significantly more likely to die before discharge (22.2%) compared with non-neuro-COVID patients (14.4%).

Many applicants referenced an early interest and exposure to neurology, often describing their first courses in neurology sparking their desire to learn more about how the brain works.

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The chief medical officer of Scholar Rock discussed the upcoming phase 3 trial for apitegromab in SMA and whether a successful trial would warrant FDA submission.

An overview of the diagnosis of spinal muscular atrophy, including prevalence, age of onset, and the role of genetic testing.

James Wymer, MD, provides a case overview of a 38-year-old female diagnosed with spinal muscular atrophy.