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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What Clinicians Need to Know About Fibrodysplasia Ossificans Progressiva
Mona Al Mukaddam, MD, Director of the Penn Bone Center at the University of Pennsylvania, discusses fibrodysplasia ossificans progressiva (FOP). FOP is a disorder in which skeletal...
Radiopharmaceutical Treatment for Neuroendocrine Tumors
Erik Mittra, MD, PhD, Professor of Diagnostic Radiology Oregon Health and Science University, discusses neuroendocrine tumors. A neuroendocrine neoplasm is a type of neuroendocrine...
Patient Perspective: Diagnostic Journey and Challenges of Lipodystrophy
Christine Coppini, patient with lipodystrophy, discusses her diagnostic journey and challenges she faces living with a rare disease. Lipodystrophies are rare metabolic disorders...
Case Report: Patient With Congenital Adrenal Hyperplasia
Christine Eliazo, Medical Student at Nova Southeastern University, discusses a case report on a patient with congenital adrenal hyperplasia (CAH). CAH is a genetic condition...
Survey Finds Wide-Ranging Impact of X-Linked Hypophosphatemia (XLH) on Patient Experiences
Findings highlight the burden of XLH on personal finances and out-of-pocket costs, overall health—physical, mental, and social—and access to expert care. XLH Community Impact Survey: Effects...
FDA Approves Sanofi’s Rilzabrutinib for Treatment of Patients with Chronic ITP
The U.S. Food and Drug Administration (FDA) has approved Wayrilz (rilzabrutinib) for the treatment of adults with persistent or chronic immune thrombocytopenia (ITP) who have had insufficient...
Education Campaign for Thyroid Eye Disease
Margarita Ochoa-Maya, MD, Medical Director of the Rare Disease Business Unit at Amgen, discusses the TEDucation campaign for thyroid eye disease (TED). TED is a rare autoimmune disease...
Naxitamab Combination Therapy for 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 naxitamab...
FDA Approves Updated REMS for IgA Nephropathy Treatment
The U.S. Food and Drug Administration (FDA) has approved the updated Risk Evaluation and Mitigation Strategy (REMS) for Filspari (sparsentan) for the treatment of IgA nephropathy (IgAN). IgAN is a...
Diagnosing and Treating a Patient With Castleman Disease
Robin Williams, MD, Pediatric Hematology Oncologist at M Health Fairview Masonic Children’s Hospital, discusses her experience diagnosing and treating a patient with Castleman disease (CD). ...
Mental Health in Patients With Acromegaly
Lori Bulpett, Manager of Patient Advocacy at Chiesi Global Rare Diseases, discusses mental health in patients with acromegaly. Acromegaly is a hormonal disorder that results from the...
FDA Approves Papzimeos (zopapogene imadenovec) for Recurrent Respiratory Papillomatosis
The U.S. Food and Drug Administration (FDA) has approved Papzimeos (zopapogene imadenovec-drba) for the treatment of adults with recurrent respiratory papillomatosis (RRP). RRP is a rare viral...
SMAshing My Limits
Tracey Dawson, PhD, SVP, U.S. Therapeutic Area Head of Neuroscience at Novartis, discusses the SMAshing My Limits campaign. Spinal muscular atrophy (SMA) is a group of genetic...
Recordati’s Presentations at ENDO 2025
Mario Maldonado, MD, Global Head of Clinical Development for Endocrinology at Recordati Rare Diseases, discusses Recordati’s presentations at ENDO 2025. LINC6 Clinical Trial LINC 6 is...
Atumelnant for the Treatment of Congenital Adrenal Hyperplasia
Alan Krasner, MD, Chief Endocrinologist at Crinetics Pharmaceuticals, discusses atumelnant for the treatment of patients with congenital adrenal hyperplasia (CAH). CAH is a genetic...
Disparities Between Biochemical Control and Symptom Burden in Patients With Acromegaly
Jason Crompton, PharmD, Global Medical Director of the Acromegaly Program at Chiesi Rare Diseases, discusses disparities between biochemical control and symptom burden in patients with acromegaly....
Recordati Rare Disease Initiatives
Mohamed Ladha, President and General Manager for Recordati Rare Diseases North America, discusses the company’s rare disease initiatives. The U.S. branch of Recordati was established...
FDA Approves Donidalorsen for Treatment of Hereditary Angioedema (HAE)
The U.S. Food and Drug Administration has approved Dawnzera (donidalorsen) for prophylaxis treatment to prevent attacks in patients ages 12 years and older with hereditary angioedema (HAE). HAE is a...
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...
Reproduction and Pregnancy in Lysosomal Disorders
Best practices to manage pregnancy and other reproductive issues associated with lysosomal disorders (LDs)
Recent Videos
TETON-2 Findings in Treatment of Idiopathic Pulmonary Fibrosis #podcast #raredisease
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Highlights from The Liver Meeting 2026 | The CheckRare Brief
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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
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
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.
View the full
Phase 3 OASIS Study Shows Promise of Oral Olorofim for Invasive Aspergillosis
Results of the Phase 3 OASIS Study of Oral Olorofim
UCB Study Highlights the Burden of Ocular Myasthenia Gravis
CheckRare 102 minutes ago