Rare Disease Focus: Neurology/Nervous System Diseasess +++ +++ +++

CLINICAL PERSPECTIVES Pre-Diagnostic Symptoms and Time-to-Diagnosis in MLD https://checkrare.com/pre-diagnostic-symptoms-and-time-to-diagnosis-in-mld/ Laura Adang, MD, PhD, Assistant Professor at Perelman School of Medicine at University of Pennsylvania, discusses a cohort study conducted analyzing pre-diagnostic symptoms and time-to-diagnosis in patients with metachromatic leukodystrophy (MLD). Symptom and Treatment Burden in Fabry Disease https://checkrare.com/symptom-and-treatment-burden-in-fabry-disease/ Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. Positive Data on Fenfluramine for the Treatment of CDKL5 Deficiency Disorder https://checkrare.com/positive-data-on-fenfluramine-for-the-treatment-of-cdkl5-deficiency-disorder/ Amélie Lothe, PhD, Head of Global Medical Community of Rare Epilepsies at UCB, discusses positive data on fenfluramine for the treatment of CDKL5 deficiency disorder (CDD). Glioblastoma Overview https://checkrare.com/glioblastoma-overview/ Erin Dunbar, MD, founding physician of the Brain Tumor Center and Director of Neuro-Oncology at Piedmont Atlanta Hospital, discusses the current landscape of glioblastoma. All Endpoints Met in Clinical Trials With Oveporexton for Narcolepsy https://checkrare.com/all-endpoints-met-in-clinical-trials-with-oveporexton-for-narcolepsy/ Takeda has announced positive data from two phase 3 clinical trials of oveporexton in patients with narcolepsy type 1 (NT1). Lumryz for Hallucinations in Patients With Narcolepsy https://checkrare.com/lumryz-for-hallucinations-in-patients-with-narcolepsy/ Michael J. Thorpy, MD, Professor of Neurology at Albert Einstein College of Medicine, discusses positive results in study of Lumryz (sodium oxybate) for hallucinations in patients with narcolepsy. Delpacibart Braxlosiran in Patients With Facioscapulohumeral Muscular Dystrophy https://checkrare.com/delpacibart-braxlosiran-in-patients-with-facioscapulohumeral-muscular-dystrophy/ Mike Flanagan, PhD, Chief Scientific Officer at Avidity Biosciences, discusses topline results from study testing delpacibart braxlosiran (del-brax) in patients with facioscapulohumeral muscular dystrophy and the newly initiated phase 3 trial. Implications of Nipocalimab Approval on Pediatric Patients With Myasthenia Gravis https://checkrare.com/implications-of-nipocalimab-approval-on-pediatric-patients-with-myasthenia-gravis/ Jonathan Strober, MD, Director of the Neuromuscular Clinic and Professor of Pediatrics and Neurology at the University of California at San Francisco, discusses the implications of nipocalimab’s approval to treat pediatric patients with myasthenia gravis (MG). New Data From EMBARK Study in Patients With Duchenne Muscular Dystrophy Receiving Gene Therapy https://checkrare.com/new-data-from-embark-study-in-patients-with-duchenne-muscular-dystrophy-receiving-gene-therapy/ Aravindhan Veerapandiyan, MD, Pediatric Neuromuscular Neurologist at Arkansas Children’s Hospital, discusses new data from the EMBARK part 2 study of Elevidys (delandistrogene moxeparvovec) in patients with Duchenne muscular dystrophy (DMD). Riliprubart for the Treatment of CIDP https://checkrare.com/riliprubart-for-the-treatment-of-cidp/ Claudia Sommer, MD, Professor of Neurology at the University of Würzburg, Germany, and Alex Seluzhytsky, MD, Senior Global Medical Director at Sanofi, discuss data on riliprubart for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).