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APN-1607 Gets Fast Track Designation, ALS Agent BIIB105 Terminated, ION582 Safe in Angelman Syndrome
Neurology News Network. for the week ending May 25, 2024. [WATCH TIME: 4 minutes]

The professor and chair of rehabilitation medicine at UT Health San Antonio talked about the newly published guidelines for the assessment and management of spasticity in patients with neurological conditions. [WATCH TIME: 5 minutes]

The double-blind, placebo-controlled trial, which includes 2 dosing regimens of COYA 302, is expected to have topline data reported later this summer.

Dyne aims to pursue expedited approval pathways for DYNE-251, leveraging dystrophin as a surrogate biomarker.

DYNE-101 demonstrated dose-dependent splicing correction, with the 5.4 mg/kg cohort showing a 27% mean correction at 3 months.

The phase 3 REACH trial investigating Fulcrum Therapeutics’ losmapimod in patients with facioscapulohumeral muscular dystrophy remains on track with topline data anticipated in the fourth quarter of 2024.

Stephanie Richardson, MS, CCC-SLP, an outpatient lead speech-language pathologist at Brooks Rehabilitation, discussed the unique aspects of speech therapy and the pivotal role it plays in helping patients with swallowing and other related functions.

Larimar Therapeutics anticipates interim data from the open label extension study assessing nomlabofusp in patients with Friedreich Ataxia in the fourth quarter of 2024.

ION582 Improves Cognition, Motor Function, and Communication in Phase 1/2 Trial of Angelman Syndrome
ION582, delivered intrathecally into the cerebral spinal fluid with a lumbar puncture, resulted in significant improvements in cognition, fine and gross motor skills, and expressive communication.

In honor of Chronic Demyelinating Polyneuropathy Awareness Month, held May 2024, get caught up on some of the latest news in CIDP, with data updates and expert insights all in one place from the NeurologyLive® team.

APN-1607 is undergoing a global phase 3 trial to evaluate its efficacy as an early diagnostic biomarker for progressive supranuclear palsy.

Here's some of what is coming soon to NeurologyLive® this week.

Avidity Biosciences anticipates to provide new cohort data from the phase 1/2 EXPLORE44 trial assessing AOC 1044 in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the second half of 2024.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on Huntington disease.

BIIB105, an antisense oligonucleotide, failed to demonstrate impact on outcomes of function, breathing, and strength, in addition to no impact on neurofilament light.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 17, 2024.

Solid Biosciences is expecting to provide initial safety data on SGT-003 from the first couple of pediatric patients with Duchenne muscular dystrophy enrolled in the phase 1/2 INSPIRE Duchenne trial in mid-2024.

Treatment with rozanolixizumab was safe among patients with CIDP, resulted in reduced immunoglobulin levels as expected, but did not have an impact on efficacy.

Cure SMA introduces tools aiding in SMA clinical trial navigation and participation empowerment.

Lindsey Lee Lair, MD, a neurologist and vice president of clinical development at Biohaven Pharmaceuticals, provided insight on an ongoing pivotal trial assessing the impacts of taldefgrobep alfa, a myostatin inhibitor, in patients with SMA.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on cluster headache.

A recent study recognized anti-DAGLA autoantibodies in the cerebrospinal fluid of a small group of patients, suggesting that it could be a potential biomarker for diagnosing cerebellar ataxia.

Neurology News Network. for the week ending May 11, 2024. [WATCH TIME: 4 minutes]

Avidity Biosciences is anticipated to initiate its phase 3 HARBOR trial assessing AOC 1001 as a treatment for patients with myotonic dystrophy type 1 in the second quarter of 2024.