Clinical Insights
Efficacy of Efgartigimod To Treat Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): ADHERE Trial
Hans Katzberg, MD, Assistant 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 demyelinating polyneuropathy (CIDP). CIDP is...
Key Features of PLGD-1
Angela Zhu, MD, Ophthalmologist and Clinical Assistant Professor, Pediatric & Adult Cornea/Cataract/External Diseases, at Bascom Palmer Eye Institute in Miami, describes some of the key features of plasminogen deficiency and why ophthalmologists are often the healthcare providers who begin the process toward diagnosis.
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).
Narcolepsy Breakthroughs and Emerging Therapies
Emmanuel Mignot, MD, PhD, is one of the pioneers in the study of narcolepsy and served a vital role in discerning the genetic cause of this rare and underdiagnosed disorder. Dr. Mignot, Professor of Sleep Medicine in the Department of Psychiatry and Behavioral Sciences, Stanford University, recently shared his thoughts on his work with CheckRare.
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Accelerated Approval Granted to Beqalzi (Sonrotoclax) for Mantle Cell Lymphoma
The US Food and Drug Administration (FDA) has granted accelerated approval to Beqalzi (sonrotoclax) for the treatment of adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL) after at...
Two-Year Results of Crenessity (Crinecerfont) in the Treatment of Congenital Adrenal Hyperplasia
Richard J. Auchus, MD, PhD, Professor of Internal Medicine and Pharmacology at the University of Michigan Medical School, discusses two-year results of Crenessity (crinecerfont) in the treatment of...
Ecopipam as a Treatment for Tourette Syndrome: Phase 3 Clinical Trial Results
Kinga Tomczak, MD, PhD, Program Director of the Tic Disorders and Tourette Syndrome Program at Boston Children’s Hospital and Assistant Professor of Neurology at Harvard Medical School, discusses...
Bizengri (Zenocutuzumab) Approved for NRG1 Fusion-Positive Cholangiocarcinoma Under the FDA’s National Priority Voucher Program
The US Food and Drug Administration (FDA) has approved Bizengri (zenocutuzumab) for the treatment of adults with NRG1 fusion-positive cholangiocarcinoma. Cholangiocarcinoma is a rare, aggressive...
FDA Expands Indication of Vyvgart (Efgartigimod Alfa) for Adults With Seronegative Myasthenia Gravis
The US Food and Drug Administration (FDA) has approved Vyvgart (efgartigimod alfa) and Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase) for the treatment of adult patients who have anti-MuSK-Ab...
New Follow-Up Data of KYV-101 Treatment for Patients With Myasthenia Gravis
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses new follow-up data on KYV-101 treatment for patients with myasthenia gravis (MG). ...
Diagnosis and Management of Plasminogen Deficiency
Amol Sura, MD, Foster Center for Ocular Immunology, Dept of Ophthalmology, Duke University, Durham, North Carolina, discusses the diagnosis and management of plasminogen deficiency, a rare condition...
8-Week Follow-Up Data of RAP-219 for the Treatment of Focal Onset Seizures
William W. Motley, MD, RAP-219 Program Leader at Rapport Therapeutics, discusses follow-up data of RAP-219 for the treatment of focal onset seizures. Focal onset seizures refer to...
GEMZ Clinical Trial of Fenfluramine in Patients With CDKL5 Deficiency Disorder
Brian Moseley, MD, Senior Medical Director at UCB, discusses the GEMZ clinical trial of fenfluramine in patients with CDKL5 deficiency disorder (CDD). CDD is a rare genetic disorder...
CareCompass: Online Tool for Caregivers of Dravet and Lennox-Gastaut Syndrome
Colin Lake, Head of Digital Business Transformation Neurology at UCB, discusses CareCompass, an online tool for caregivers of patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS)....
FDA Approves Extended Release Ruxolitinib for Three Rare Hematologic Disorders
The US Food and Drug Administration (FDA) has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of three rare hematologic conditions. The indications include the treatment...
CureFest 2026
Dena Sherwood, mother to a neuroblastoma survivor and Founder of Arms Wide Open Childhood Cancer Foundation, discusses CureFest 2026. Ms. Sherwood and her family founded the Arms Wide...
Positive Results with Cemdisiran, an RNAi Mediated Therapy, in Patients With Myasthenia Gravis
Tuan Vu, MD, Professor, Department of Neurology at the University of South Florida, discusses positive results of cemdisiran in patients with generalized myasthenia gravis (gMG). MG is...
Effects of Rodatrisat Ethyl on Activity in Patients With Pulmonary Arterial Hypertension
A study published in the Annals of the American Thoracic Society analyzed results from the ELEVATE-1 clinical trial testing the effects of rodatristat ethyl on daily activity in patients with...
An Overview of Systemic Mastocytosis
Dareen D. Siri, MD, FAAAAI, FACAAI is a board-certified allergist and immunologist practicing at Midwest Allergy Sinus Asthma, based in central Illinois. She talked with CheckRare about a unique,...
Accelerated Approval Granted to Gene Therapy for Rare Genetic Hearing Loss
The US Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni (lunsotogene parvec) for the treatment of patients with otoferlin (OTOF)-related hearing loss. The indication...
Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated With Velaglucerase Alfa
A study published in the Journal of Clinical Medicine evaluated the improvement of bone mineral density in patients with type 1 Gaucher disease (GD1) treated with velaglucerase alfa. GD1 is the most...
Daybue (Trofinetide) Stix: New Formulation of Treatment for Rett Syndrome
Arthur Beisang, MD, Department of Pediatrics at Gillette Children's Specialty Healthcare in Saint Paul, Minnesota, discusses Daybue (trofinetide) Stix, a new formulation of the treatment for Rett...
Quality of Life in Patients With Pulmonary Arterial Hypertension
A study recently published in Health and Quality of Life Outcomes analyzed quality of life in patients with pulmonary arterial hypertension (PAH). PAH is a rare condition affecting the heart and...
Effect of Uplizna (Inebilizumab) on Ocular Manifestations in Myasthenia Gravis
Richard J. Nowak, MD, global principal MINT investigator and director of the Myasthenia Gravis Clinic at Yale University, discusses a post-hoc analysis of Uplizna (inebilizumab) on the effect of...
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Join the World Orphan Drug Congress USA 2026! Connect with leaders, regulators, and patient advocates to advance rare disease therapies, clinical trials, and patient access strategies from June 9–11 in Boston.
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METEOROID Clinical Trial of Enspryng (Satralizumab) in Patients With MOGAD
New Trial Comparing Rituximab With Targeted Therapies in Patients With NMOSD
ADAPT SERON Clinical Trial of Efgartigimod IV in Seronegative Generalized Myasthenia Gravis
DUET Clinical Trial of Xywav in Patients With Narcolepsy
PLDG-1 is underdiagnosed, but with increasing awareness, and the availability of effective treatment, diagnosis and management of these patients can be improved today.
Learn more at https://checkrare.com/diagnosis-and-management-of-plasminogen-deficiency/
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Data Presented on Myasthenia Gravis at the 2026 American Academy of Neurology Meeting
Upcoming Regulatory Decision for LNTH-2501 in the Imaging of Neuroendocrine Tumors
Results From the ACUITY Clinical Trial in Patients With Acute Optic Neuritis
PLDG-1 is underdiagnosed, but with increasing awareness, and the availability of effective treatment, diagnosis and management of these patients can be improved today.
Link in bio.
#CheckRare #PLGD #RareGenetic #RareDisease
Systemic Mastocytosis: Recognition, Diagnosis, and Clinical Management
Improving Patient Outcomes and Quality Of Life in AVP-D
AVP-D Management Advances
AVP-D Traditional Management














METEOROID Clinical Trial of Enspryng (Satralizumab) in Patients With MOGAD
CheckRare May 19, 2026 2:21 pm