Phase 2 Data Show ACE-083 Was Safe, Improved Muscle Function in Charcot-Marie-Tooth Disease Type 1
May 22nd 2022Despite good tolerability with few drug-related treatment emergent AEs and increased muscle volume, the failure to provide clinically relevant improvements in muscle function led to the discontinuation of ACE-083 for CMT in 2020.
Progress in Neurogenerative Disease Drug Development: Gregory A. Rippon, MD, MS
May 19th 2022The chief medical partner of neurology, ophthalmology, and internal medicine at Genentech shared his perspective on the steps the field has taken to improve clinical trials and therapies for neurodegenerative diseases such as Alzheimer disease. [WATCH TIME: 3 minutes]
Reactions to FDA Advisory Panel Decision on AMX0035: Justin Klee; Josh Cohen, BSc
May 19th 2022The co-CEOs and co-founders of Amylyx Pharmaceuticals discussed their immediate thoughts to the recent FDA AdComm meeting, which voted against evidence of efficacy for AMX0035 in ALS. [WATCH TIME: 3 minutes]
NeuroVoices: Mark Lew, MD, on the Validation of Tears as a Biomarker for Parkinson Disease
May 18th 2022The director of the Division of Movement Disorders at the USC Keck School of Medicine provided insight on the next steps to justify whether reflex tears are a reliable biomarker for Parkinson disease.
AlzoSure Predict Test Shows Predictive Ability for Alzheimer Disease Years Prior to Diagnosis
May 17th 2022The blood test from Diadem SpA, which quantifies the AZ 284 peptide as readout of U-p53AZ, showed a significantly higher AUC in both time-independent and time-dependent prognostic analyses than gold standard testing.
Adding Experience to the National Health Institute Council: Amy Brin, MSN, MA, PCNS-BC
May 17th 2022The chief executive officer of the Child Neurology Foundation provided insight on her new position at the National Health Institute’s National Advisory Neurological Disorders and Stroke Council. [WATCH TIME: 4 minutes]
Cognitive Functioning and Motor Difficulties Identified in Spinal Muscular Atrophy Type 3
May 16th 2022When compared with healthy controls, patients with SMA type 3 performed worse on tests that assessed executive function, language, and visuospatial abilities, suggesting that intrinsic brain pathology may exist in these patients.
AUPN 2022 Spring Chairs Session: Simulations for Training in Neurology
May 16th 2022David G. Standaert, MD, PhD; Wolfgang Muhlhofer, MD; and Latisha Sharma, MD, share their experience in applying simulation-based learning in Neurology and will discuss the current state and future potential of this approach. [WATCH TIME: 1 hour, 25 minutes]
Advocacy’s Part in the Progress in ALS: Matthew B. Harms, MD
May 16th 2022In light of ALS Awareness Month, the associate professor of neurology at Columbia University discussed the ways in which advocacy has provided a platform for patients and improved conversations in care. [WATCH TIME: 6 minutes]