The US Food and Drug Administration (FDA) has approved Orzeyful (oveporexton) for the treatment of adults with narcolepsy type 1 Narcolepsy is a chronic neurological disorder characterized by an inability of the brain to control sleep-wake cycles. Patients with...
Neurology/Nervous System Diseases
Rare neurological disorders are diseases of the brain, spine, and autonomic nervous system. This section provides overviews of several of those conditions, as well news, clinical trial updates, and expert opinions form leading neurologists.
LOTUS Study in Patients With Rett Syndrome Treated With Daybue (Trofinetide)
Ryan Bucco, PharmD, Vice President of Medical Affairs in Rare Disease at Acadia, discusses the LOTUS study in patients with Rett syndrome treated with Daybue (trofinetide).
Initiation of Phase 3 FORZETTO Clinical Trial Testing Zeleciment Rostudirsen in Duchenne Muscular Dystrophy
Doug Kerr, MD, PhD, MBA, Chief Medical Officer of Dyne Therapeutics, discusses the initiation of the phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
August Is SMA Awareness Month
August is SMA Awareness Month, a time dedicated to increasing understanding of spinal muscular atrophy (SMA) and supporting the individuals and families impacted by this rare genetic neuromuscular condition.
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Results From the PROGRESS Study of Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva
Joanne Quan, MD, Chief Medical Officer of Mirum Pharmaceuticals, discusses results from the PROGRESS study of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). FOP...
Prader-Willi Syndrome: Diazoxide Choline Extended Release Improves Hyperphagia
Evelien Gevers, PhD, MD, Consultant Pediatrician and Reader in Endocrinology and Diabetes at Barts Health NHS Trust, discusses effects of diazoxide choline extended release (DCCR) on hyperphagia in...
Treatment With Xywav (Low Sodium Oxybate) in Patients With Narcolepsy and Idiopathic Hypersomnia
Logan Schneider, MD, Adjunct Clinical Associate Professor of Psychiatry and Behavioral Sciences at Stanford University, discusses treatment with Xywav (low sodium oxybate) in patients with sleep...
Spinal Muscular Atrophy: The Changing Definition of Success
Spinal muscular atrophy (SMA) has undergone a remarkable transformation over the past decade. Drs. Nancy Kuntz, Alicia Henriquez, and Angela Lek discuss how advances in disease-modifying therapies...
Safety and Efficacy of Subcutaneous Efgartigimod PH20 in Ocular Myasthenia Gravis
Carolina Barnett-Tapia, MD, Neuromuscular Neurologist and the University of Toronto, discusses the safety and efficacy of subcutaneous efgartigimod PH20 in ocular myasthenia gravis (oMG). ...
Impact of Vyvgart (Efgartigimod Alfa) Approval for Patients with Seronegative Myasthenia Gravis
Jeff Guptill, MD, Neuromuscular Clinical Development Lead at Argenx, discusses the impact of the Vyvgart (efgartigimod alfa) approval on the myasthenia gravis (MG) community to treat patients with...
Preferences and Perceptions of Acute Seizure Medications
Kerrie-Anne Ho, PhD, Patient Preference Lead at UCB, discusses preferences and perceptions of acute seizure medications. Data from interviews with people with seizures (PwS) and...
Efgartigimod in Juvenile Generalized Myasthenia Gravis
Abigail Schwaede, MD, Assistant Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses results on intravenous efgartigimod in juvenile generalized myasthenia...
Maternal and Neonatal Outcomes of Chenodeoxycholic Acid Treatment in Pregnant Women With Cerebrotendinous Xanthomatosis
A study published in the Journal of Clinical Lipidology evaluated maternal and neonatal outcomes of chenodeoxycholic acid (CDCA) treatment during gestation in women with cerebrotendinous...
Results from the ADAPT SERON Clinical Trial of Efgartigimod IV in Patients With Seronegative Generalized Myasthenia Gravis
James Howard Jr., MD, Professor of Neurology at the University of North Carolina at Chapel Hill, discusses results from the ADAPT SERON clinical trial of efgartigimod IV in patients with...
METEOROID Clinical Trial of Enspryng (Satralizumab) in Patients With MOGAD
Michael Levy, MD, PhD, Neuroimmunologist and Associate Professor at Harvard Medical School, discusses results from the METEOROID clinical trial of Enspryng (satralizumab) in patients with myelin...
New Trial to Compare Rituximab With Targeted Therapies in Patients With Neuromyelitis Optica Spectrum Disorder
Sumaira Ahmed, Founder and Executive Director of The Sumaira Foundation, discusses her organization and the BEST-NMOSD clinical trial for patients with neuromyelitis optica spectrum disorder...
Arginine Vasopressin Deficiency (AVP-D) Overview
Christopher Romero, MD, a pediatric endocrinologist at Mount Sinai Medical Center, New York City, and Associate Professor of Pediatrics at the Icahn School of Medicine at Mount Sinai discusses...
DUET Clinical Trial of Xywav in Patients With Narcolepsy
Logan Schneider, MD, Adjunct Clinical Associate Professor of Psychiatry and Behavioral Sciences at Stanford University, discusses the DUET clinical trial of Xywav (low sodium oxybate) in patients...
Data Presented on Myasthenia Gravis at the 2026 American Academy of Neurology Meeting
Omar Sinno, MD, US Medical Strategy Lead for Rare Disease at UCB Pharma, discusses data presented on myasthenia gravis (MG) at the 2026 American Academy of Neurology meeting. MG is a...
Itvisma Gene Therapy for Patients With Spinal Muscular Atrophy
John Day, MD, PhD, Director of Neuromuscular Medicine at Stanford University, discusses the development of Itvisma gene therapy for patients with spinal muscular atrophy (SMA). SMA is...
Upcoming Regulatory Decision for LNTH-2501 in the Imaging of Neuroendocrine Tumors
Mauro Cives, MD, Associate Professor of Medical Oncology at the University of Bari, Italy, discusses the upcoming regulatory decision for LNTH-2501 to improve imaging of neuroendocrine tumors...
Results From the ACUITY Clinical Trial in Patients With Acute Optic Neuritis
Pablo Villoslada, MD, PhD, Founder and Medical Director of Accure Therapeutics and Head of Pathogenesis and New Therapies MS at IDIBAPS in Hospital Clínic in Barcelona, discusses results from the...
Efficacy of Efgartigimod To Treat Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): ADHERE Trial
Hans Katzberg, MD, Professor of Neurology at the University of Toronto and Neurologist at Toronto General Hospital, discusses post hoc analyses from the ADHERE clinical trial in chronic inflammatory...
Primary Analysis of KYV-101 for the Treatment of Stiff Person Syndrome
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses primary analysis of KYV-101 for the treatment of stiff person syndrome (SPS). SPS is a...













Initiation of FORZETTO Clinical Trial Testing Zeleciment Rostudirsen in Duchenne Muscular Dystrophy
CheckRare August 6, 2026 2:48 pm