Marco Meglio

Building on positive phase 1a data, ALX-001 demonstrated target engagement of mGluR5 receptor and maintained a safe profile across multiple doses.

Monepantel, a novel, potent, inhibitor of the mTOR pathway, was safe in patients with ALS, resulting in stabilized ALS Functional Rating Scale scores.

The delivery device is prefilled with the prescribed dose of Acthar Gel in 40- or 80-unit versions, and may help administration of the appropriate subcutaneous dose of Acthar Gel.

Comprised of more than 400 patients with SMA, results showed that wanting to be treated with all possible DMTs was the most common reason for combination therapy.

Amanda Peltier, MD, a professor of neurology at Vanderbilt University Medical Center, spoke on current care needs for adults with neuromuscular disorders and the realistic ways to enhance clinical care going forward.

GA Depot, composed of extended-release microspheres administered intramuscularly, typically every 28 days, was expected to result in fewer injection site reactions than other glatiramer acetate products.

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 Purple Day and epilepsy awareness.

These finding suggest that even immune cells in the brain can play a significant role in the progression of neurodegenerative diseases like FA.

Over the coming weeks, Amylyx will undergo heavy discussions with regulators and community members to decide the next best steps for AMX0035 after the agent failed to meet its primary end point in its latest study.

The FDA will discuss TRAILBLAZER-ALZ 2, a large-scale phase 3 study that assessed the effects of donanemab in 1736 patients with early-stage Alzheimer disease over a 76-week period.

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Alcibiades Rodriguez, MD. [LISTEN TIME: 19 minutes]

The real-world, 3-year study is expected to include 30 males with SMA on risdiplam, an SMN2 splicing modifier, to assess fertility-related outcomes.

The cofounders and cochief executive officers at Amylyx Pharmaceuticals provided commentary on the incorporation of AMX0035 for patients with ALS and the upcoming PHOENIX confirmatory trial.

Median time to loss of ambulation for golodirsen-treated patients was 1968 days vs 1092 days for external control patients.

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.

Phase 2 study analysis of Roche's fenebrutinib reveals similar safety profiles across multiple autoimmune indications, including relapsing multiple sclerosis.

Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.

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 Alzheimer disease and dementia.

Using a 4-point score change on Symbol Digits Modalities Test, more than 80% of patients had either sustained improvement or no sustained change after 2 years of treatment with cladribine.

GA Depot, which remains under review by the FDA, showed a safe and tolerable profile in an additional 52-week extension period.

These data, presented at 2024 ACTRIMS Forum, highlight the impacts neurofilament light has on patients with multiple sclerosis irrespective of race or ethnicity.

The conversations behind cerebroprotection in stroke have begun to get louder, with a pipeline that includes several different approaches to treatment.

ATH-1105 protected various cell culture systems from glutamate-mediated toxicity and its pathological alterations, which include mitochondrial dysfunction, apoptotic signaling, and TDP-43 mis-localization, among others.

The antisense oligonucleotide will continue to be assessed in a large-scale phase 3 study, CARDIO-TTRansform, which will have data read out in 2025.

The primary result showed a highly significant effect of edaravone dexborneol on good functional outcome, defined by scores of 0 (no symptoms) or 1 (some symptoms) on the standard modified Rankin scale assessed at 90 days postrandomization.

Repetitive transcranial magnetic stimulation, a more widely used symptomatic treatment, was able to improve several outcomes for patients with cerebellar ataxia, especially at high frequencies.

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 sleep disorders and hypersomnias.