October 15th 2024
A new analysis of the phase 3 PROPEL study showed that switching to cipaglucosidase alfa/miglustat therapy enhanced outcomes among patients with late-onset Pompe disease.
Get expert-driven insight and coverage of key data and research straight from the conference floor in Savannah.
Cases and Conversations™: Applying Best Practices to Prevent Shingles in Your Practice
October 16, 2024
Register Now!
Mastering MS: Translating Evidence into Optimal Management Plans
View More
Advances In Treating Migraine in Your OB/GYN Practice: Navigating Treatment Paradigms to Improve Patient Care
View More
Patient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis - Current Treatment and Emerging Con...
November 12, 2024
Register Now!
Recognizing Rett Syndrome Early to Improve Long-term Management Outcomes
View More
2024 Neuromuscular Summit
November 20, 2024
View More
5th Annual International Congress on the Future of Neurology®
View More
Clinical Consultations™: Managing Depressive Episodes in Patients with Bipolar Disorder Type II
View More
Key Takeaways for MS Providers Regarding Major Medicare Changes Expected in 2025
October 11th 2024Sarah Anderson, PharmD, NBC-HWC, senior director of clinical resources and programming at National MS Society, talked about the significant updates under the Inflation Reduction Act to come in 2025 for patients with multiple sclerosis.
FDA Removes Partial Hold for Myotonic Dystrophy Agent AOC 1001
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.
Duchenne Gene Therapy Shows No Alteration of Cardiac Outcomes in Phase 3 EMBARK Study
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.
Exploring the Impact of Circadian Rhythms on Glial Function and Alzheimer Disease
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.
PTC to Submit NDA for Vatiquinone in Friedreich Ataxia Following Positive Long-Term Extension Data
Overall, vatiquinone-treated patients demonstrated persistent effects on disease progression over a 144-week long-term period, leading to a future regulatory submission.