Clinical Insights
Results from the MajesTEC-9 and CARTITUDE-4 Clinical Trials in Patients With Relapsed/Refractory Multiple Myeloma
Roberto Mina, MD, Assistant Professor at Winship Cancer Institute at Emory University, discusses results from the MajesTEC-9 and CARTITUDE-4 clinical trials in patients with relapsed/refractory multiple myeloma (RRMM). MM is a rare blood cancer...
Going Beyond Lab Values: Cognitive Impairment in Patients With Immune Thrombocytopenia
David Kuter, MD, DPhil, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, discusses cognitive impairment in patients with immune thrombocytopenia (ITP).
FDA Approves Jideytro (Zidesamtinib) for Adults With ROS1-Positive Non-Small Cell Lung Cancer
The US Food and Drug Administration (FDA) has approved Jideytro (zidesamtinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who have received a prior ROS1 kinase inhibitor.
Results From the PROGRESS Study of Zilurgisertib in Patients With Fibrodysplasia Ossificans Progressiva
Joanne Quan, MD, Chief Medical Officer of Mirum Pharmaceuticals, discusses results from the PROGRESS study of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP).
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Ongoing Research for Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay
Sonia Gobeil, co-founder of Ataxia of Charlevoix-Saguenay Foundation, discusses their organization and ongoing research for autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS). ...
Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring
Progressive Familial Intrahepatic Cholestasis (PFIC): Diagnosing, Treating, Monitoring Patrick McKiernan, MD and Nadio Ovchinsky, MDThis educational program, hosted by Patrick McKiernan, MD,...
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP)
Consider Rare: Suspecting and Diagnosing Fibrodysplasia Ossificans Progressiva (FOP) Ellen Elias, MD and Christiaan Scott, MDFibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic...
Newborn Screening Update Following RFK’s Removal of Advisory Committee
Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval. As noted by Mr. Suhr,...
FDA Approves Myasthenia Gravis Treatment Nipocalimab
The U.S. Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG). The approval is for patients, 12 years of age and older, with...
Recent Data Highlights Benefits of Sepiapterin in Patients With PKU
Nicola Longo, MD, PhD, Professor and Chief of Division of Clinical Genetics at the University of California, Los Angeles, discusses data presented at the 2025 ACMG Annual Clinical Genetics Meeting...
FDA Approves Gene Therapy for Epidermolysis Bullosa
The U.S. Food and Drug Administration (FDA) has approved Zevaskyn (prademagene zamikeracel or pz-cel) for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (EB). EB...
FDA Approves Hepatocellular Carcinoma Combination Therapy
The U.S. Food and Drug Administration (FDA) has approved nivolumab plus ipilimumab for first-line therapy in adult patients with unresectable or metastatic hepatocellular carcinoma (HCC). HCC is a...
The Impact of Hemophilia on Women and Girls
Shellye Horowitz, a patient with hemophilia and advocate, discusses the impact of hemophilia on women and girls. Hemophilia Hemophilia is a bleeding disorder that slows the blood clotting...
Neuroblastoma: Overview, Curie Scores, and Treatment Options
Greg Yanik, MD, Clinical Professor at University of Michigan's C.S. Mott Children's Hospital, provides an overview of this rare childhood cancer, goals of therapy, using the Curie score, and...
ATLG Versus ATG in Graft-Versus-Host Disease
A new study published in American Journal of Hematology compared two treatment options for Graft versus host disease (GVHD). Graft Versus Host Disease GVHD is a rare complication that can occur...
Neuroblastoma: Evan Foundation
Gavin Lindberg details his family’s experience with neuroblastoma, provides advice to families, and discusses the EVAN Foundation. ...
Efficacy of Nipocalimab To Treat Myasthenia Gravis: Updates From Vivacity-MG3 Trial
Sindhu Ramchandren, MD, Executive Medical Director, Neuroscience and Disease Cluster Lead for Neuroimmunology and Neuromuscular Disorders at Johnson & Johnson, discusses updated results from the...
Effect of Increased IgG Levels on Prognosis in Primary Biliary Cholangitis
A recent study published in Liver International analyzed patients with primary biliary cholangitis (PBC) who presented with higher IgG levels at diagnosis. Primary Biliary Cholangitis PBC is...
Safety and Efficacy of Zilucoplan to Treat Myasthenia Gravis: Results from the RAISE Trial
Michael Weiss, MD, Department of Neurology, University of Washington Medical Center, discusses results from the RAISE clinical trial testing the safety and efficacy of zilucoplan in patients with...
Efficacy of Atrasentan To Treat Patients With IgA Nephropathy
Richard Lafayette, MD, Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, discusses the recent approval of Vanrafia (atrasentan)...
Digital Voice Analysis as a Biomarker of Acromegaly
A recent study published in The Journal of Clinical Endocrinology & Metabolism analyzed the use of digital voice analysis as a biomarker of acromegaly. Acromegaly is an endocrine disorder...
New FDA-Approved C3 Glomerulopathy (C3G) Treatment Targets Underlying Cause of Disease
Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses the recent U.S. Food and Drug Administration (FDA) approval of iptacopan as the first and only treatment of...
Clinical Outcomes in Peripheral Ulcerative Keratitis
A recent study published in the American Journal of Ophthalmology observed clinical outcomes in patients with peripheral ulcerative keratitis (PUK). Peripheral Ulcerative Keratitis PUK is an...
FORWARD-53 Clinical Trial in Duchenne Muscular Dystrophy Shows Promise
Paul Bolno, MD, President and Chief Executive Officer of Wave Life Sciences, discusses positive results from the FORWARD-53 clinical trial evaluating WVE-N531 in patients with Duchenne muscular...
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FDA Approves New Narcolepsy Treatment
FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-W...
Rare Endocrine Exchange: Why This Conversation Matters
Emmanuel Mignot, MD, PhD, is a pioneer in the study of narcolepsy and served a vital role in discerning the genetic cause of this rare and underdiagnosed disorder.
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FDA Approves Multiple Myeloma Treatment, Denies NETs Treatment | The CheckRare Brief
FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-W...
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What is SMA? Learn all about this rare disease during SMA Awareness Month 🎗️💜
Learn more at the link in our bio.
#CheckRare #SMAAwarenessMonth #SMA #SpinalMuscularAtrophy #RareMusculoskeletal #RareNeurology
Patient Perspective: Diagnostic Journey With IgA Nephropathy
Addressing Patient Experiences With IgA Nephropathy
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#CureFest2026
🎗️We are 1 month away from the 2026 CureFest for Childhood Cancer event, hosted by the Arms Wide Open Childhood Cancer Foundation
🗓️September 18-20 in Washington, DC
Register at the link in our bio.
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Rare Endocrine Exchange Episode 1: Why Rare Endocrine Matters












FDA Approves Multiple Myeloma Drug; FDA Denies Neuroendocrine Drug; Safety Concerns For Prader-W...
CheckRare August 20, 2026 2:57 pm