Clinical Insights
Mirum’s Livmarli Achieves Primary Endpoint in Phase 3 EXPAND Study
Mirum Pharmaceuticals recently announced positive topline results from its phase 3 EXPAND study, evaluating Livmarli (maralixibat) for cholestatic pruritus in patients six months and older with ultra-rare cholestatic liver diseases. Jennifer Vittorio, MD, an EXPAND...
Generalized Myasthenia Gravis: AANEM 2026 Highlights
The American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM) held its 2026 annual meeting September 29–October 1 in Orlando. CheckRare focused its meeting coverage on generalized myathesthenia gravis (gMG), and its treatment and management.
Miv-cel Shows Durable Results in Stiff Person Syndrome and Myasthenia Gravis
Naji Gehchan, MD, MBA, Chief Medical and Development Officer at Kyverna Therapeutics, discusses one-year data showing sustained benefits of KYV-101 (mivocabtagene autoleucel ; Miv-cel) in stiff person syndrome (SPS), along with longer-term follow-up data in generalized myasthenia gravis (gMG).
How Bispecific Antibodies Are Changing Multiple Myeloma Treatment
Nisha Joseph, MD, Associate Professor in the Department of Hematology and Medical Oncology at the Emory University School of Medicine, discusses how bispecific antibodies are changing multiple myeloma treatment.
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FDA Approves Oral Treatment for Arginine Vasopressin Deficiency
The US Food and Drug Administration (FDA) has approved Desmoda (desmopressin acetate) oral solution for the management of arginine vasopressin deficiency (AVP-D). AVP-D is a rare neuroendocrine...
Survey of Clinical Guidelines for MPS II
Kim Stephens, DBA, Executive Director of the Muenzer MPS Research & Treatment Center, discusses a survey of clinical guidelines for mucopolysaccharidosis II (MPS 2; Hunter syndrome). ...
Accelerated Approval Granted to Loargys (pegzilarginase) for the Treatment of ARG1 Deficiency
The US Food and Drug Administration (FDA) has granted accelerated approval to Loargys (pegzilarginase-nbln) for the treatment of patients ages 2 years and older with arginase 1 deficiency (ARG1-D)....
FDA Approves Venetoclax Combination Therapy for Adults With Chronic Lymphocytic Leukemia
The US Food and Drug Administration (FDA) has approved Venclexta (venetoclax) in combination with acalabrutinib therapy for untreated adult patients with chronic lymphocytic leukemia (CLL). CLL is...
Phase 1/2 Results of Tividenofusp Alfa in Patients With MPS II
Joseph Muenzer, MD, PhD, Pediatric Biochemical Geneticist at University of North Carolina Chapel Hill, discusses results from a phase 1/2 study of tividenofusp alfa in patients with...
From Seizures to Sequencing: How Genetic Testing Is Changing Epilepsy Care
Sophia Ceulemans, MS, Medical Science Liaison at GeneDx offered a high-level overview of the problem of unexplained epilepsy and the rapidly advancing field of genetic testing for this challenging...
Targeting HRD and BRCA: The Expanding Role of PARP Inhibitors in Ovarian Cancer Treatment
Bradley J. Monk, MD, Medical Director, Florida Cancer Specialists and Research Institute, West Palm Beach, and Founder of GOG Partners, discusses the clinical advances in the treatment of ovarian...
Real-World Outcomes of Arimoclomol in Patients With Niemann Pick Type C
Caroline Hastings, MD, Pediatric Hematology Oncology, Professor of Pediatrics at the University of California, San Francisco, discusses real-world outcomes of arimoclomol in patients with Niemann...
Age-Stratified Clinical Burden of Post-Allogeneic HSCT Complications in Hurler Syndrome (MPS IH)
Ali Mohajer, PhD, Qral Group, discusses the age-stratified burden of post-allogeneic hematopoietic stem cell transplantation (post-allo-HSCT) complications in Hurler syndrome (MPS IH). MPS IH...
MajesTEC-9 Clinical Trial of Teclistamab in Patients With Multiple Myeloma
Roberto Mina, MD, Associate Professor at Winship Cancer Institute of Emory University, discusses the Majestec-9 clinical trial of Tecvayli (teclistamab) in patients with multiple myeloma (MM)....
Long-Term Outcomes From the PIONEER Study in Patients With Indolent Systemic Mastocytosis
Tsewang Tashi, MD, Hematologist at the Huntsman Cancer Institute at the University of Utah, discusses long-term data from the PIONEER clinical trial examining the use of avapritinib in patients with...
Using Artificial Intelligence to Analyze Castleman Disease Histopathology
Robert S. Ohgami, MD, PhD, Professor of Pathology, University of Utah, and Founding Vice President and Chief Medical Director, ARUP Institute for Research and Innovation, discusses the utilization...
Elritercept’s Effect on Transfusion Independence in Patients With Myelodysplastic Syndromes
Lynette Chee, PhD, Hematologist at The Royal Melbourne Hospital/ Peter MacCallum Cancer Centre, discusses elritercept’s effect on transfusion independence (TI) in patients with myelodysplastic...
February 28 Is Rare Disease Day
February 28 Is Rare Disease Day! Rare Disease Day, observed on the last day of February every year, is a reminder of the challenges faced by those living with a rare disease....
52-Week Results From the VERIFY Clinical Trial of Rusfertide in Patients With Polycythemia Vera
Andrew T. Kuykendall, MD, VERIFY Lead Investigator and Associate Member in the Department of Hematology at Moffitt Cancer Center, discusses 52-week results from the VERIFY clinical trial testing...
Committee for Medicinal Products for Human Use Recommendation for Sotatercept for the Treatment of Pulmonary Arterial Hypertension
Marius Hoeper, MD, Respiratory Physician at Hannover Medical School, Germany, discusses a recent regulatory update by the Committee for Medicinal Products for Human Use (CHMP) of the European...
Positive Topline Results from a Study Testing Nipocalimab in Patients With Systemic Lupus Erythematosus
Leonard L. Dragone, MD, PhD, Disease Area Leader in Autoantibody and Rheumatology, Johnson & Johnson Innovative Medicine, discusses positive topline results from a study of nipocalimab in...
Approval of Nerandomilast Tablets To Treat Patients With Progressive Pulmonary Fibrosis
Shervin Assassi, MD, Director, Division of Rheumatology at McGovern Medical School, discusses the approval of Jascayd (nerandomilast) tablets for treatment of patients with progressive pulmonary...
Results from the XTEND-ed Clinical Trial Evaluating Efanesoctocog Alfa for the Treatment of Patients With Hemophilia A
Lynn Malec, MD, Versiti Blood Research Institute and Medical College of Wisconsin, discusses results from the XTEND-ed clinical trial evaluating efanesoctocog alfa for the treatment of patients with...
Neuroblastoma
Neuroblastoma is a rare childhood cancer, but it is the most common extracranial solid tumor in children. It is a neuroendocrine tumor that originates in neuroblasts or neural crest progenitor...
Mogamulizumab in Danish Patients With CTCL
Lena Specht, MD, DMSc, talks with CheckRare about her Danish study supporting the use of mogamulizumab in Danish patients with CTCL.
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Anna Kole, Global Patient Engagement Lead for Myasthenia Gravis at UCB, discusses the findings from a mixed-methods study examining the experiences and unmet needs of people living with ocular myasthenia gravis (oMG) and explains how patient perspectives could improve clinical research and Read morecare.
View the full interview at https://checkrare.com/ucb-study-highlights-the-burden-of-ocular-myasthenia-gravis/
What should you know about myasthenia gravis?
From understanding the underlying disease mechanisms to exploring FDA-approved treatment options and ongoing clinical research, staying informed is essential for the rare disease community.
The Myasthenia Gravis Disease Learning CenterRead more brings together educational information and resources to help patients, caregivers, and healthcare professionals better understand this complex condition.
Explore the learning center: https://checkrare.com/myasthenia-gravis/
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UCB Study Highlights the Burden of Ocular Myasthenia Gravis
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Steven Nathan, MD, MBBCh, FCCP, Director of the Advanced Lung Disease and Lung Transplant Program at Inova Fairfax Hospital, discusses findings from a post-hoc analysis of the phase 3 TETON-2 study, presented at the CHEST 2026 Annual Meeting.
View the full interview at Read morehttps://checkrare.com/inhaled-treprostinil-reduces-multiple-disease-progression-events-in-ipf/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
What's the latest research in rare liver conditions?
Get updates from The Liver Meeting 2026 and more on this week's episode of The CheckRare Brief at https://checkrare.com/highlights-from-the-liver-meeting-2026-the-checkrare-brief/
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Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
View the full interview at Read morehttps://checkrare.com/phase-3-oasis-study-shows-promise-of-oral-olorofim-for-invasive-aspergillosis/
Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven (UZ Leuven) in Belgium and Principal Investigator of the OASIS study, discusses positive phase 3 trial results evaluating oral olorofim in patients with invasive aspergillosis.
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Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
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