November 20th 2024
Over a 12-month treatment period, patients on deferiprone demonstrated decreased blood ferritin and hippocampal QSM but caused accelerated cognitive decline and increased regional brain atrophy.
Get direct access to expert insight and perspective, and all the coverage of the key data presentations, straight from the conference floor in Madrid.
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
Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
View More
Medical Crossfire®: Understanding the Advances in Bipolar Disease Treatment—A Comprehensive Look at Treatment Selection Strategies
View More
Burst CME: Optimizing Therapy in Parkinson’s Disease
View More
Neurogene Reports Serious Adverse Event in Phase 1/2 Rett Study of Gene Therapy NGN-401
The company has decided to pause enrollment in the high-dose cohort of its phase 1 clinical trial for NGN-401 after a participant experienced a serious treatment-related adverse event.
New Phase 2 SYNAPSE-CMT Study to Test Effects of NMD670 in Charcot-Marie-Tooth Disease Type 1 and 2
The phase 2 trial is expected to include 80 adults with genetically confirmed CMT who will be followed for a 21-day treatment period, using changes in functional assessments as the primary outcome.
New Postmarketing Registry to Evaluate Real-World Safety and Patient Experience With Omaveloxolone
The SKYCLARYS PASS registry, an international observational cohort study, aims to include approximately 300 omaveloxolone-naïve patients participating in the Friedreich’s Ataxia Global Consortium's UNIFIED natural history study.
CHMP Gives Positive Opinion for Lecanemab Following Re-Examination of Data
The change in opinion was after reconsidering the totality of data from the phase 3 Clarity AD trial, a large-scale study of lecanemab featuring more than 1500 patients with early-stage Alzheimer disease.
VQ-101 Show Promise in Interim Phase 1 Data for GBA-Parkinson Disease Treatment
November 16th 2024According to the company, VQ-101 is the first small molecule to show over 75% activation of the lysosomal enzyme GCase in humans, with phase 1 results in healthy volunteers indicating tolerability and exposure levels that support once-daily dosing.
GAD-Related Parkinson Gene Therapy AAV-GAD Demonstrates Efficacy in Topline Findings
November 14th 2024Initial findings showed significant improvements from baseline in the disease-specific, patient-reported quality of life scores in both the high and low dose AAV-GAD groups, with no significant change in the sham group at 26 weeks.
FDA Clears Icobrain Aria, First AI Tool for Safer ARIA Detection in Alzheimer Treatment
The software, designed using thousands of brain MRI scans, is used for automated detection and severity grading of ARIA-E and ARIA-H for safety monitoring of new Alzheimer disease therapies.
FDA Approves Expanded Age Indication for Nerivio Migraine Device in Pediatric Patients
November 14th 2024Nerivio, a nondrug option for acute migraine attacks and prevention of migraine with or without aura, has been FDA-approved for several years in adolescents aged 12 and older as well as adults.
FDA Grants Accelerated Approval to PTC Therapeutics’ Gene Therapy for AADC Deficiency
November 13th 2024A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.
Why Trump and Musk's Alliance Could Launch America's Neurotech Moonshot
Neal K. Shah, CEO at CareYaya Health Technologies, shared his perspective on how the new administration could unleash innovation in neurotech, reduce regulatory barriers with Elon Musk’s involvement, and accelerate innovations that could improve neurological care for those with Alzheimer and dementia.