Neuromuscular

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.

The published data provided preliminary evidence that AMX0035 engages Alzheimer disease pathology and pathways of neurodegeneration, synaptic function, gliosis, and oxidative stress.

Findings from a recent study analyzing 3 cohorts suggested that certain proteins in the cerebrospinal fluid may have potential as biomarkers or therapeutic targets in progressive supranuclear palsy.

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.