Clinical Insights
Z-Basivarsen Shows Functional Improvements in One-Year Data for Myotonic Dystrophy Type 1
Laurie Gutmann, MD, professor of neurology at Indiana University, discusses data showing functional improvements with zeleciment basivarsen (z-basivarsen; DYNE-101) in patients with myotonic dystrophy type 1. Understanding Myotonic Dystrophy Type 1...
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).
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).
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Data on Paltusotine for the Treatment of Acromegaly
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses data on the efficacy and safety of paltusotine for the treatment of acromegaly. Acromegaly is an...
Tips to Belong to the Rare Diseases Clinical Research Network (RDCRN)
Eva Morava-Kozicz, MD, PhD, Mayo Clinic in Rochester, MN shares some tips for a rare disease group to be part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National...
Impact of Approval of Pegcetacoplan for Rare Kidney Diseases
Carla Nester, MD, University of Iowa Stead Family Children's Hospital and lead investigator of the VALIANT study, discusses the impact of the recent approval of pegcetacoplan (Empaveli) for...
Clinical Experience With Iptacopan for Treatment of PNH
Jamie Koprivnikar, MD, Hematologist Oncologist at Hackensack University Medical Center, discusses her clinical experience with iptacopan for the treatment of paroxysmal nocturnal hemoglobinuria...
Linvoseltamab in the Treatment of Relapsed/Refractory Multiple Myeloma
Joshua Richter, MD, Associate Professor of Medicine at the Tisch Cancer Institute, Director of Multiple Myeloma at the Blavatnik Family- Chelsea Medical Center at Mount Sinai, discusses the recent...
Evaluating Fenfluramine in Patients With Lennox-Gastaut Syndrome
Amelie Lothe, PhD, Global Medical Community Head for Rare Epilepsies at UCB, discusses recent data evaluating fenfluramine in patients with Lennox-Gastaut syndrome. Developmental and...
New Staging Tool Available for Healthcare Providers Treating Patients With CTCL
Pamela Blair Allen, MD, MSc, Hematologist, discusses the new CTCL Staging Tool on PROBEinCTCL.com. Cutaneous T-cell Lymphomas (CTCL) are rare, heterogeneous types of non-Hodgkin T-cell...
Outpatient Administration of Naxitamab in Patients With Neuroblastoma
Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses outpatient...
Immune Checkpoint Inhibition in Patients With Epstein-Barr Virus-Associated Gastric Cancer
James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses outcomes of immune checkpoint inhibition in patients with Epstein-Barr virus-associated gastric cancer (EBVaGC)....
Unmet Medical Needs in Rare Diseases
Tobias Hagedorn, Secretary of the European Society for PKU (ESPKU), discusses unmet medical needs in rare disease communities. Phenylketonuria (PKU) is a genetic metabolic disorder...
FDA Approves Dordaviprone for Rare Type of Glioma
The U.S. Food and Drug Administration (FDA) has approved Modeyso (dordaviprone) for the treatment of patients with diffuse midline glioma with a H3 K27M mutation. Glial tumors are a type of brain...
Results From Amivanatamab Clinical Trial for Metastatic Colorectal Cancer
Dirk Arnold, MD, PhD, Director of the Asklepios Tumorzentrum Hamburg, discusses results from the OrigAMI-1 clinical trial in left-sided RAS/BRAF wild-type metastatic colorectal cancer (mCRC). ...
Erdheim-Chester Disease: Diagnosis and Treatment Options
Skand Shekhar, MD, Principal Investigator at the National Institutes of Health (NIH), discusses Erdheim-Chester disease diagnosis and treatment options. ECD is a rare blood cancer...
FDA Approves Concizumab for Hemophilia A or B Without Inhibitors
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab) injection as a once-daily prophylaxis treatment to prevent or reduce frequency of bleeding episodes in patients ages 12...
The ARISE Cancer Consortium
Javier Oesterheld, MD, Division Chief of the Cancer and Blood Disorders Program at Levine Children’s Hospital and Founder and Executive Director of the ARISE Cancer Consortium, discusses the...
Risk Stratification in Patients With mIDH Glioma
James Hamrick, MD, Chairman of the Caris Precision Oncology Alliance, discusses age in risk stratification in patients with mutant isocitrate dehydrogenase (mIDH) glioma. mIDH is a...
The FDA Approves Sepiapterin for Treatment of Phenylketonuria
The U.S. Food and Drug Administration (FDA) has approved Sephience (sepiapterin) for the treatment of phenylketonuria (PKU). PKU is a rare genetic metabolic disorder that increases the body's...
Advancements in the Treatment of Myasthenia Gravis
Henry J. Kaminski, MD, Professor of Neurology at The George Washington University, Lead of the Myasthenia Gravis Rare Disease Network (MGNET), explains “Myasthenia Gravis: The Future Is Here,” an...
The FDA Approves Pegcetacoplan for Treatment of Rare Kidney Disorders (C3G and IC-MPGN)
The U.S. Food and Drug Administration (FDA) has approved Empaveli (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis...
FDA Approves Lonapegsomatropin for Treatment of Adults With Growth Hormone Deficiency
The U.S. Food and Drug Administration (FDA) has approved Skytrofa (lonapegsomatropin-tcgd; TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency...
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 16 minutes ago