Clinical Insights
New Formulation of Berotralstat Approved for Adults and Adolescents With Hereditary Angioedema
The US Food and Drug Administration (FDA) has approved the oral pellet formulation of once-daily Orladeyo (berotralstat) for prophylactic therapy in patients ages 12 years and older with hereditary angioedema (HAE). HAE is a rare condition characterized by recurrent...
FDA Approves Gazyva to Reduce Relapse Risk in Patients With Idiopathic Nephrotic Syndrome
Andrea Callaway, president and founder of The Nephrotic Syndrome Foundation, discusses the FDA approval of Gazyva (obinutuzumab) for idiopathic nephrotic syndrome (INS) and what the approval means for affected families.
FDA Approves New Formulation of Sjögren’s Disease Treatment
The US Food and Drug Administration (FDA) has approved Evoxac (cevimeline HCI) oral solution to address dry mouth in patients with Sjögren’s disease (SjD).
FDA Approves Enspryng, the First At-Home Treatment Option for Thyroid Eye Disease
The US Food and Drug Administration (FDA) has approved Enspryng (satralizumab) for the treatment of adults with thyroid eye disease (TED).
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Panel Discussion: The High Cost of Rare Diseases
Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their...
Cutaneous T-Cell Lymphoma: Natural History and the Need for Better Diagnostic and Treatment Efficacy
Henry Wong, MD, PhD, Dermatologist at the San Diego Veterans Hospital and the University of California San Diego (UCSD), discusses cutaneous T-cell lymphoma (CTCL). CTCL is a rare group of...
The Value of the Rare Diseases Clinical Research Network (RDCRN)
Henry J. Kaminski, MD, Professor of Medicine at The George Washington University describes the value of being in the Rare Diseases Clinical Research Network (RDCRN). Dr. Kaminsky leads the Dr....
The FDA Approves Avatrombopag to Treat Young Children with ITP
The U.S. Food and Drug Administration (FDA) has approved Doptelet (avatrombopag) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients one year and older who have had...
Combination PD-1 Antibody and GPRC5D Antibody Therapy in Patients With Relapsed/Refractory Multiple Myeloma
Paula Rodríguez-Ortero, MD, PhD, Hematologist at the University of Navarra, discusses results of PD-1 inhibitor combination therapy in patients with multiple myeloma (MM). MM is a bone...
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...
Rilzabrutinib in the Treatment of IgG4-Related Diseases
John Stone, MD, Professor of Medicine at Harvard Medical School and Edward A. Fox Chair in Medicine at Massachusetts General Hospital, discusses data on rilzabrutinib in the treatment of...
Symptom and Treatment Burden in Fabry Disease
Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. Fabry disease is a rare lysosomal storage disease characterized by a...
Andembry (Garadacimab) for the Treatment of Hereditary Angioedema
Timothy Craig, DO, Tenured Professor of Medicine, Pediatrics, and Biomedical Sciences at Penn State University, Principal Investigator of the VANGUARD trial, discusses the recent approval of...
Recent Clinical Trial Data Testing Inebilizumab to Treat IgG4-Related Diseases
Matthias Löhr, MD, PhD, Professor of Gastroenterology & Hepatology at Karolinska Institutet, Stockholm, Sweden, discusses recent data from the MITIGATE clinical trial of inebilizumab for the...
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). CDD is a...
Chronic Myeloid Leukemia: Diagnosis and Treatment
Michael Mauro, MD, Director of the Chronic Myeloid Leukemia Program at Memorial Sloan Kettering Cancer Center, discusses the diagnosis and treatment of chronic myeloid leukemia (CML). ...
Effect of Elafibranor on Fatigue in Patients With PBC
Sandra Silvestri, MD, PhD, Chief Medical Officer at Ipsen, discusses the effect of elafibranor on fatigue in patients with primary biliary cholangitis (PBC). PBC is a chronic,...
Unmet Needs of Patients With wAIHA
Ann Leon, PharmD, Global Medical Affairs Leader of Benign Hematology at Johnson & Johnson, discusses the unmet needs of patients with warm autoimmune hemolytic anemia (wAIHA). ...
Promising Results for Avapritinib in Observational Trial of Patients With Indolent Systemic Mastocytosis
Researchers from Berlin, Germany and Beirut, Lebanon have confirmed in a prospective, observational study that patients with uncontrolled symptoms of indolent systemic mastocytosis (ISM) can improve...
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. Glioblastoma...
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). NT1 is a rare, chronic neurological disorder characterized by excessive...
Marfan Syndrome: Diagnosis and Management
David Liang, MD, PhD, Cardiologist at Hoag Hospital, discusses the diagnosis and management of Marfan syndrome. Marfan syndrome is a rare genetic disorder characterized by problems in...
Mezagitamab in Patients With Immune Thrombocytopenia
David Kuter, MD, DPhil, Director of Clinical Hematology at Massachusetts General Hospital, discusses recent results of a trial of mezagitamab in patients with immune thrombocytopenia (ITP). ...
Effects of Ciltacabtagene Autoleucel in Patients With Multiple Myeloma
Surbhi Sidana, MD, Hematologist and Associate Professor at Stanford University, discusses effects of ciltacabtagene autoleucel (cilta-cel) in patients with multiple myeloma (MM). MM is...
Investigating Results From the MAVORIC Trial in Patients With CTCL
H. Miles Prince, MD, MBBS, describes results of an investigation of previous results from the MAVORIC trial in patients with CTCL.
Recent Videos
The Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026 | The CheckRare Brief
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New research is helping advance the understanding and treatment of rare kidney diseases.
The VALIANT study evaluated pegcetacoplan, a targeted complement therapy, in patients with C3G and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).
Explore the study Read moreand hear more about the importance of advancing treatment options for patients, including children.
Read the full story: https://checkrare.com/c3g-and-the-pediatric-valiant-study/
The Need for Earlier Pulmonary Hypertension Screening in Interstitial Lung Disease
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/
#MyastheniaGravis #RareDiseaseEducation #HealthcareProfessionals #ClinicalResearch #PatientEducation
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
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
Living With Ocular Symptoms of Myasthenia Gravis
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/
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026
Inhaled Treprostinil Reduces Multiple Disease Progression Events in IPF
Highlights from The Liver Meeting 2026 | The CheckRare Brief
Interim PHINDER Analyses in Interstitial Lung Disease
CheckRare 17 minutes ago