Marco Meglio

Jonas Hannestad, MD, PhD, chief medical officer at Gain Therapeutics, provided clinical perspective on promising data from a phase 1 first-in-human study of GT-02287, an investigational therapy for Parkinson disease with or without a GBA1 mutation.

Over an 18-month period, patients showed strengthened scores on Rasch-built Overall Disability Scale, a patient-reported outcome measure of activity and social participation.

In this segment, the panel discussed adult-onset 5q spinal muscular atrophy (SMA), highlighting its frequent underdiagnosis and subtle symptoms such as cramps, fasciculations, and sports intolerance.

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 Alzheimer disease.

The discussion highlighted the need for national or local protocols, insurance approvals, and close collaboration with families to optimize patient outcomes.

In this discussion, experts provide key considerations in the process of newborn screening for SMA, noting the need for developing local protocols and understanding the limitations of screening infants early in life.

HNSA-5487 exhibited lower pre-treatment anti-drug antibody levels and reduced ADA responses compared to imlifidase, indicating a favorable immunogenicity profile and strong potential for redosing, with no serious adverse events observed.

AOC 1001, also known as del-desiran, has shown promising results in improving muscle strength and reducing myotonia in patients with DM1, with patients even reporting better daily activities like hand grip strength.

In this episode, experts discuss groundbreaking updates in spinal muscular atrophy diagnosis, emphasizing the importance of early intervention and newborn screening.

After 52 weeks of treatment, no significant differences were found in cardiac MRI measures such as ejection fraction, end diastolic volume, or circumferential strain between the two groups.

The decision follows positive data from multiple trials, showing significant improvement in both cardiac function and muscle performance in patients with DMD.

Alexander disease, a rare leukodystrophy estimated to occur in 1 in 1 million patients in the United States, has no FDA-approved treatments to date.

Erik Musiek, MD, PhD, a professor of neurology at Washington University in St. Louis, provided clinical insight on a presentation from ANA 2024 highlighting the intricate relationship between circadian rhythms, glial activation, and neuroinflammation in Alzheimer disease.

In this episode, our panel of experts delves into the latest updates on spinal muscular atrophy diagnosis, focusing on the critical role of newborn screening, early intervention, and evolving classification methods for improved patient outcomes.

No serious adverse events were reported, and the therapy demonstrated a favorable safety profile over the 5-year study period.

Overall, vatiquinone-treated patients demonstrated persistent effects on disease progression over a 144-week long-term period, leading to a future regulatory submission.

RAG-18 is an saRNA delivered to muscle tissue to increase UTRN expression and compensate for DMD defects regardless of the location of mutation on DMD gene.

Patients aged 2-21 receiving apitegromab showed clinically meaningful motor function improvements, with a favorable safety profile consistent with long-term data, as Scholar Rock prepares for U.S. and EU regulatory submissions in Q1 2025.

Michelle Bravo, MD, an assistant professor of clinical neurology at the University of Miami, provided commentary on a rare case report of a subdural hematoma linked to spontaneous intracranial hypotension caused by a refractory CSF leak.

Over half of the patients (56.7%) discontinued at least one class of acute migraine medication within 12 months post-erenumab initiation.

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 insomnia.

The phase 2 study, expected to include 575 patients with acute migraine, will use percentage of participants with freedom from pain and freedom from the most bothersome symptom as the coprimary end points.

Over a 12-month period, patients showed sustained improvements in disease progression through valbenazine, regardless of concomitant antipsychotic treatment.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Daniel Ontaneda, MD, PhD. [LISTEN TIME: 27 minutes]

The KINETIC 2 study did not show statistically significant improvement in essential tremor symptoms compared to placebo, leading to the termination of its development for this condition.

This trial aims to explore the role of the noradrenergic system in behavioral and cognitive outcomes and could lead to new therapeutic strategies for managing PSP.

ION464 is designed to inhibit the production of the alpha-synuclein protein, aiming to reduce the accumulation of this protein in the brain, which is believed to be a key factor driving neurodegeneration in MSA and Parkinson disease.

The consultant neurologist and senior lecturer at St. Georges Hospital in London, provided commentary on the diagnosis, management, and future research around neuropsychiatric symptoms of Parkinson disease.