NeurologyLive

🚨 #BREAKING: The FDA has approved levacetylleucine (Aqneursa; IntraBio Inc) for the treatment of ataxia in adults and pediatric patients with ataxia-telangiectasia (A-T) weighing at least 15 kg. The approval marks the first FDA-approved treatment indicated specifically for A-T and is based on data from the phase 3 IB1001-303 trial. Read More: https://hubs.li/Q04xVNHP0

🎙️ Episode 175 of Mind Moments, "Advancing the Ways of Treating Cluster Headache," is out now! 🤕 Neurologists Jennifer Fokas, MD, and Minali Nigam, MD, both of Northwestern Medicine, discussed the substantial burden of chronic cluster headache and the diagnostic overlap with migraine that often delays proper care. 🚶They walked through the cranial autonomic symptoms and attack patterns that help distinguish cluster headache, along with acute and preventive treatment approaches, including high-flow oxygen, subcutaneous sumatriptan, bridge therapies, high-dose verapamil, and select use of lithium. 🎧 Listen to the full episode: https://hubs.li/Q04xRGqK0 ❇️ Spotify: https://hubs.li/Q04xRGnf0 #ClusterHeadache #HeadacheMedicine #Neurology #MindMoments #Podcast

🧬 A phase 2 trial of the investigational RIPK1 inhibitor SAR443820 did not demonstrate a functional benefit in amyotrophic lateral sclerosis (#ALS) The HIMALAYA trial randomized 305 adults with ALS to SAR443820 20 mg twice daily or placebo for 24 weeks. At week 24: 📊 ALSFRS-R change: −6.73 with SAR443820 vs −6.32 with placebo 📉 Between-group difference: −0.41 points (95% CI, −1.71 to 0.88) SAR443820 was also associated with a higher rate of adverse events and treatment discontinuations, with elevated hepatic enzymes the most common reason for discontinuation The findings highlight the challenge of translating biologically compelling targets, including pathways involved in neuroinflammation and regulated cell death, into meaningful clinical benefit in ALS Read the full NeurologyLive report for the trial findings and clinical context: https://hubs.li/Q04xK-sK0 #Neurology #Neurodegeneration #ClinicalTrials #DrugDevelopment

Ahead of the 4th Annual Updates in Neurology CME Symposium on October 9, NeurologyLive spoke with presenter Hannah Kopinsky, MD, about challenges in diagnostic criteria in MS care, shifts in disease-modifying therapies and more. Read the Q&A in NeurologyLive: https://lnkd.in/e_jiMp8k

In a recent NeurologyLive feature, Jacqueline Becker, PhD Assistant Professor of Medicine (General Internal Medicine) and Director of Neurocognitive Health at the Icahn School of Medicine at Mount Sinai, discusses the cognitive symptoms associated with Long COVID, including persistent “brain fog” that can affect attention, processing speed, and everyday functioning. A key takeaway for physicians: listen to what patients are experiencing and recognize when additional evaluation may be needed. Dr. Becker discusses how neuropsychological evaluation can help when cognitive symptoms persist, interfere with daily life, or aren’t captured by routine screening. She also explores emerging approaches to cognitive rehabilitation and ongoing research into the long-term neurological effects of COVID-19. Listen to the full interview with MJH Life Sciences® in our comments below.

🚨 #BREAKING: The FDA has approved Ultragenyx's gene therapy UX111 (Fayuvi) for the treatment of Sanfilippo syndrome type A, making it the first approved disease-modifying therapy for the condition. Read More 👉 https://hubs.li/Q04xPg-y0

😴 Advances in hypersomnia diagnosis and treatment could reshape how clinicians approach excessive daytime sleepiness 🗣️ In a NeurologyLive interview, KuangHua Guo, MD, PhD, sleep specialist at Northwestern Medicine, discussed the emergence of orexin agonists and their potential role in the treatment landscape for narcolepsy and other hypersomnia disorders 🧠 Guo also highlighted the importance of: • Ruling out secondary causes of hypersomnia before pursuing specialized testing • Using objective sleep testing to help distinguish conditions such as idiopathic hypersomnia and narcolepsy type 2 • Monitoring patients closely as newer orexin-targeting therapies enter clinical practice Read the full interview for a look at the evolving diagnostic and therapeutic landscape: https://hubs.li/Q04xF0YQ0 #Neurology #SleepMedicine #Hypersomnia #Narcolepsy #MedicalEducation

🧠 🆕 phase 4 data add to the growing evidence highlighting the challenges of treating pain in Parkinson disease 🚫 In the OCEAN trial, once-daily opicapone 50 mg did not demonstrate statistical superiority over placebo for fluctuation-related pain at 24 weeks 📊 KPPS domain 3 scores decreased by 8.8 points with opicapone vs 9.3 points with placebo (P = .7940) 🔗 Read the full analysis from NeurologyLive: https://hubs.li/Q04xD_yn0 #ParkinsonDisease #Neurology #MovementDisorders #ClinicalTrials #Pain

iPSC models are reshaping neuropathy research. But does that mean animal models should go? Ahmet Hoke, MD, PhD, of Johns Hopkins makes the case for keeping them in the pipeline in Episode 1 of Gray Areas, NeurologyLive's new series tackling neurology's toughest questions. ⚗️ https://hubs.li/Q04xvSL30

September is Muscular Dystrophy Awareness Month. NeurologyLive rounds up 8 investigational therapies to watch across DMD, DM1, and FSHD, from RNA-based approaches and cell therapy to DUX4 suppression. 💪 #MuscularDystrophy #Neurology https://hubs.li/Q04xvSr50