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). oMG is a neuromuscular disease characterized by autoantibody...
Autoimmune / Autoinflammatory Disorders
Disease overviews, clinically relevant perspectives, and news about important research in rare autoimmune and auto-inflammatory disorders.
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 anti-MuSK-Ab positive, anti-LRP4-Ab positive, and/or triple seronegative MG.
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 gravis (gMG).
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 seronegative generalized myasthenia gravis (gMG).
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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...
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...
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...
Outcomes of Pegcetacoplan Treatment in Adolescents With C3G and IC-MPGN
A recent publication in the Clinical Journal of the American Society of Nephrology presented outcomes of pegcetacoplan treatment in adolescents with C3 glomerulopathy (C3G) and primary immune...
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...
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). ...
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...
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,...
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...
Phase 3 Results of Brepocitinib for the Treatment of Dermatomyositis
Ben Zimmer, CEO of Priovant Therapeutics, discusses results from the recent phase 3 study testing brepocitinib for the treatment of dermatomyositis (DM). DM is a rare autoimmune...
Tepezza (Teprotumumab) for the Treatment of Thyroid Eye Disease
Prem Subramanian, MD, PhD, Chief of Neuro-Ophthalmology at the University of Colorado Anschutz, discusses Tepezza (teprotumumab-trbw) for the treatment of thyroid eye disease (TED). ...
Biomarkers and Beyond: Integrating AI in Rare Disease Management
Staci Kallish, DO, Clinical Geneticist at Penn Medicine in Philadelphia, is helping to lead a new wave of innovation at the intersection of artificial intelligence (AI) and rare disease care. With a...
FDA’s Plausible Mechanism Framework and its Effect on Rare Disease Therapy Development
Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. On...
Beyond the Rash: Recognizing and Managing Systemic Mastocytosis in Clinical Practice
Systemic mastocytosis is a rare disease in which excess mast cells are produced and activated, resulting in chronic, severe, and heterogenous symptoms. CheckRare spoke with Patrick C. Foy, MD, a...
Nipocalimab Granted Fast Track Designation in Systemic Lupus Erythematosus
Richard A. Furie, MD, Chief of the Division of Rheumatology at Northwell Health, discusses nipocalimab for the treatment of systemic lupus erythematosus (SLE). SLE is an autoimmune disease...
Psychological Aspects of Rare Autoimmune and Ophthalmic Diseases
A study published in World Journal of Clinical Cases investigated the psychological aspects of rare autoimmune and ophthalmic diseases, such as mental well-being and everyday functioning. The goal...
Accelerated FDA Approval of Ex-Vivo Gene Therapy for Children with LAD-1
The US Food and Drug Administration (FDA) has granted accelerated approval to Kresladi (marnetegragene autotemcel) for the treatment of pediatric patients with severe leukocyte adhesion deficiency-1...












CEPHEUS Clinical Trial of Daratumumab Combination Therapy in Newly Diagnosed Multiple Myeloma
CheckRare June 16, 2026 4:08 pm