Neuromuscular

Zevra Therapeutics plans to immediately start its launch activities for arimoclomol, marketed as Miplyffa, which is anticipated to be commercially available in 8 to 12 weeks in the United States.

The phase 1b/2a dose escalation trial of KINE-101, conducted in Korea, includes patients with chronic inflammatory demyelinating polyneuropathy who have received at least 1 frontline treatment regimen.

Neal K. Shah, CEO of CareYaya Health Technologies, discussed a miniaturized brain-machine interface developed that could restore communication for patients with neurological disorders by translating brain activity into text.

In the study, patients with spinocerebellar ataxia treated with troriluzole demonstrated statistically significant improvements at years 1 and 2 on the treatment.

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

Neurology News Network. for the week ending September 21, 2024. [WATCH TIME: 4 minutes]

Known as QRL-101-03, the phase 1 study is expected to include 60 healthy participants, with topline results expected in the first half of 2025.

A pair of neurologists from the University of California, Irvine, provided commentary on the ways to help young investigators find their footing in research settings, and exposing them to opportunities major governmental agencies can provide. [WATCH TIME: 4 minutes]

A 28-year-old patient initially diagnosed with Guillain–Barré Syndrome presented with multiple cranial neuropathies and was later rediagnosed with chronic inflammatory demyelinating polyneuropathy.

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The general neurologist at Brigham and Women’s Hospital described the approach and mindset in teaching neurology as the field continuously evolves. [WATCH TIME: 3 minutes]

A recent study suggests that patients with chronic inflammatory demyelinating polyneuropathy lacking nerve conduction study evidence of demyelination can still respond positively to immunomodulatory therapies.

At the 2024 AUPN Annual Meeting, a pair of neurologists provided perspectives on the attributes and ethics that make a great educator, stressing passion, intellectual humility, and inclusivity, among others. [WATCH TIME: 5 minutes]

The phase 1 study of RAG-17 in patients SOD1-ALS revealed safety, tolerability, and early clinical benefits across all dose levels.

A recent study showed that an increase in anti-hepatitis E virus seroprevalence in patients with chronic inflammatory demyelinating polyneuropathy was not a consequence of intravenous immunoglobulin therapy.

New findings from the CAMPSIITE trial highlight that RGX-121 gene therapy significantly reduces cerebrospinal fluid biomarkers, suggesting its potential as a first treatment for Hunter syndrome.

Catch up on any of the neurology news headlines you may have missed over the course of August 2024, compiled all into one place by the NeurologyLive® team.

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

The chief executive officer and founder at CureDuchenne discussed efforts to bring specialized physical therapy and infrastructure support to underserved regions to improve care for patients impacted by Duchenne worldwide. [WATCH TIME: 5 minutes]

Treatment with DYNE-251 revealed dose dependent exon skipping and dystrophin expression as well as improvement in multiple functional end points in both cohorts of the phase 1/2 DELIVER trial.

Tanya, a patient who lost her mobility due to undiagnosed spinal issues, discussed her life-saving surgery for spinal meningioma, and the complex surgical process she underwent to regain her strength.

Based on the positive topline data, the company plans to submit for regulatory approval for a higher dose of nusinersen for spinal muscular atrophy treatment.

Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, talked about how the prioritization of mirdametinib, a promising treatment for both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, provided clinical perspective on some of the improvements in the everyday life and care of patients with spinal muscular atrophy.

Screening for DMD in Massachusetts is expected to begin by June 2026, which could enable earlier diagnosis and intervention.

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

Mirdametinib, a treatment in development for adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas, was granted a PDUFA target action date of February 28, 2025.

Neurology News Network. for the week ending August 31, 2024. [WATCH TIME: 4 minutes]

The FDA has granted fast track designation to [18F]PI-2620 for 3 neurodegenerative conditions, highlighting its potential to improve early diagnosis and treatment.

The submission was based on results from the phase 3 Vivacity-MG3 study, which demonstrated nipocalimab’s superiority over placebo when added to standard of care in patients with generalized myasthenia gravis.