
News and clinical perspective including CME programs focused on rare diseases. CheckRare focuses on rare and neglected diseases.
| Publishes | Daily | Episodes | 187 | Founded | 5 years ago |
|---|---|---|---|---|---|
| Number of Listeners | Categories | MedicineHealth & Fitness | |||

Dinesh Patel, PhD, CEO of Protagonist Therapeutics, discusses the New Drug Application (NDA) submission to the US Food and Drug Administration (FDA) for rusfertide to treat adults with polycythemia vera (PV).
PV is characterized by excess red blood ... more
Shunji Tomatsu, MD, PhD, Professor and Head, Nemours Children’s Health, Delaware, USA; Alessandra d’Azzo, PhD, Emerita Faculty, Genetics, St. Jude Children’s Research Hospital, Tennessee, USA; Merve Emecen Sanli, MD, Associate Professor, Department o... more
Duarte C. Barral, PhD, Associate Professor, NOVA Medical School, NOVA University of Lisbon, Portugal; Nuno Raimundo, PhD, Associate Professor, Department of Cellular and Molecular Physiology; Penn State College of Medicine, Pennsylvania, USA; Betul C... more
Oral Alpan, MD, Immunologist, Amerimmune, Virginia, USA; Svenja Keller, PhD student, University of Zurich, Switzerland; Shoshana Revel-Vilk, MD, PhD, Director, Gaucher Unit & Pediatric Hematology/Oncology Unit, Shaare Zedek Medical Center, Jerusalem,... more
Mia Horowitz, PhD, Tel Aviv University; Aitor Aguirre, PhD, Michigan State University, Michigan, USA; and Ying Sun, PhD, University of Cincinnati, discuss the use of organoid models in lysosomal disorder research and drug development.
This continuin... more
Stephan Stern, PhD, DABT, Director of Research and Development, Nanotechnology Characterization Lab (NCL), Frederick National Laboratory for Cancer Research, Maryland, USA; and Ruben Boado, PhD, Professor of Medicine, University of California at Los ... more
Behzad Najafian, MD, Professor, Department of Laboratory Medicine & Pathology, Department of Medicine at the University of Washington, Washington, USA discusses the use of artificial intelligence in identifying and managing lysosomal disorders.
This... more
Julie A. Parsons, MD
Haberfield Endowed Chair in Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine, Children's Hospital Colorado
Aurora, CO, USA
How have programs adapted to t... more
Excellent information
Good information on rare diseases
Key themes from listener reviews, highlighting what works and what could be improved about the show.
How this podcast ranks in the Apple Podcasts, Spotify and YouTube charts.
Apple Podcasts | #42 | |
Apple Podcasts | #163 |










Recent interactions between the hosts and their guests.
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This show centers on rare diseases with a strong emphasis on clinical perspectives, emerging therapies, and practical patient management. Across episodes, guests explore gene therapies for lysosomal storage disorders, early diagnosis and monitoring of LSDs, diagnostic challenges and treatment options for CIDP, and the evolving therapeutic landscape in conditions like ITP, iTTP, hemophilia, myasthenia gravis, mastocytosis, PFIC, and FOP. The hosts often highlight unmet clinical needs, the impact of diagnostics on patient outcomes, and the real-world implications of new therapies, from safety and tolerability to quality of life and access. A notable strength is the blend of specialist clinicians, researchers, and patient-centered perspectives... more
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Rare Disease Discussions launched 5 years ago and published 187 episodes to date. You can find more information about this podcast including rankings, audience demographics and engagement in our podcast database.
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Recent guests on Rare Disease Discussions include:
1. Christine Eleeson
2. Raphael Schiffmann
3. Maya Kineen
4. Nicola Longo
5. William Burns, MD
6. David Warnock
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