Neuromuscular

The chief executive officer and chief medical officer at Quince Therapeutics talked about recent findings from the phase 3 ATTeST study assessing EryDex in pediatric patients with ataxia telangiectasia.

The director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, discussed SMA Awareness Month and the enhancements to newborn screening for the disease. [WATCH TIME: 4 minutes]

EryDex did not meet its primary end point possibly because of delays in treatment reducing the number of patients who received the agent, and different treatment effects based on age.

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

DT-216P2 shows promise in treating patients with Friedreich ataxia based on positive phase 1 trial results that indicated a significant increase in frataxin mRNA levels.

A recent review showed different fluid biomarkers were involved in neurodegeneration, oxidative stress, metabolism, microRNA and novel genes in spinocerebellar ataxia type 3.

The data suggest that PrimeC's modulation of iron homeostasis may be a key mechanism in its therapeutic effect, supporting its advancement to phase 3 trials.

Researchers detected annual changes in patients with preataxic and early ataxic spinocerebellar ataxia in brain MRI imaging, clinical scores, gait parameters, and retinal thickness.

According to a recent announcement, Larimar Therapeutics reported that all 7 sites of the open-label extension study were activated for assessing nomlabofusp in Friedreich ataxia.

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

In a recent analysis, patients with chronic inflammatory demyelinating polyneuropathy who started their initial treatment later experienced a worsening of the disease course.

Neurology News Network. for the week ending August 17, 2024. [WATCH TIME: 4 minutes]

Prior to this, the FDA granted regenerative medicine advanced therapy designation to the cell therapy for refractory stiff-person syndrome.

Findings suggest that autoimmune diseases including neuromyelitis optica spectrum disorder and myasthenia gravis may also benefit from BCMA-CD19 bispecific CAR-T therapy.

In honor of Spinal Muscular Atrophy (SMA) Awareness Month, Abraham Homer, MS, the gaming technology supervisor and creative technologist at Children's Hospital Colorado, shared how virtual reality transformed care for patients with SMA.

AOC 1044, an investigational antisense oligonucleotide treatment for patients with Duchenne muscular dystrophy, has previously been granted orphan drug and fast track designation by the FDA.

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

Scholar Rock's TOPAZ phase 2 trial shows sustained motor function improvements in SMA patients treated with apitegromab over 48 months, with phase 3 results expected soon.

Long-term treatment with Clene Nanomedicine’s CNM-Au8 was associated with improved survival among patients with amyotrophic lateral sclerosis from 2 clinical trials.

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

MoMeNtum will evaluate the efficacy and safety of DNTH103, administered subcutaneously every two weeks over a 17-week period, followed by a 52-week open-label extension to collect additional safety and efficacy data.

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

In addition to showing a slowed disease progression, TPN-101-treated patients had lowered levels of key biomarkers involving neurodegeneration and neuroinflammation.

The meeting with the American Neurologic Association fosters collaboration, networking, and joint sessions, enhancing knowledge sharing among academic neurologists.

Enrollment in the dose level 2 expansion cohort is expected to complete early in Q3 2024, with initial strength and functional data anticipated in the second half of 2024.