Neuromuscular

Taldefgrobep alfa, a muscle-targeted experimental treatment potentially used in combination with other SMA therapies, will be evaluated in a cohort of 180 patients with SMA, regardless of ambulatory status or disease classification.

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Neurology News Network for the week ending July 9, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 8, 2022.

In addition to meeting primary end point of change in North Star Ambulatory Assessment, treatment with SRP-9001 resulted in statistically significant improvements in timed function tests.

Expert clinicians offer their insight on the sleep disorder landscape, the state of mental health in pediatric migraine, an inaugural movement disorders congress, a novel stroke rehabilitation tool, and psychedelics in cluster headache.

In the original panel discussion, held in March 2022, the majority of members of the committee voted that AMX0035 did not fully demonstrate sufficient efficacy as a treatment for ALS.

Catch up on any of the neurology news headlines you may have missed over the course of the last month, compiled all into one place by the NeurologyLive® team.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending July 1, 2022.

Following positive phase 2a findings, NeuroSense’s agent PrimeC continued to show a mechanistic impact on biomarkers specific to patients with amyotrophic lateral sclerosis.

In total, 100% and 93% of those in the SMN two- and three-copy cohorts achieved the primary end point of sitting independently for at least 30 seconds after 18 months of treatment.

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Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending June 24, 2022.

The decision to stop clinical testing of SRP-5051 in DMD follows a serious adverse event of hypomagnesemia in part B of the phase 2 MOMENTUM trial.

Based on the 35-week interim trial results, AstraZeneca and Ionis will seek regulatory approval for eplontersen and plan to file a new drug application with the FDA in 2022.

Both the early- and delayed-start tofersen treatment groups demonstrated reductions in SOD1 protein, the intended target for tofersen, and neurofilament, a marker of axonal injury and neurodegeneration.

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Neurology News Network for the week ending June 18, 2022. [WATCH TIME: 3 minutes]

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Mary Schroth, MD, and Jacqueline Glascock, PhD, of Cure SMA, share their perspectives on the upcoming annual meeting.

Given the importance of mitochondrial and endoplasmic reticulum function in ALS, the disruption of intracellular mitochondria-endoplasmic reticulum contacts presents yet another avenue for neuronal degeneration—perhaps the primary point of underlying dysfunction.

The approval was based on results from the phase 3 HELIOS-A study, which showed that vutrisiran met its primary end point of change in modified Neuropathy Impairment Score + 7 over a 9-month treatment period.

The approval was based on phase 2/3 findings that showed an average score of 2.32 points higher on ALS Functional Rating Scale with AMX0035 compared with placebo.

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