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Rare Research Report

RDCRN
Rare Diseases Clinical Research Network
Myasthenia Gravis
Eosinophilic Esophagitis
Global Leukodystrophy Initiative Clinical Trials Network
Consortium Of Eosinophilic Gastrointestinal Disease Researchers
Charcot-Marie Tooth Disease
Inherited Neuropathy Consortium
Metachromatic Leukodystrophy
Journal Of Allergy and Clinical Immunology
Phelan-Mcdermid Syndrome
Alexander Disease
Index Of Severity For Eosinophilic Esophagitis
Osteogenesis Imperfecta
Hereditary Hemorrhagic Telangiectasia
Primary Ciliary Dyskinesia
CMT Health Index
Air Pollution
Primary Glomerular Diseases
Clinical Trials
Developmental Synaptopathies Consortium

Rare Research Report features summaries of recent scientific publications from the Rare Diseases Clinical Research Network, which is funded by the National Institutes of Health. The network includes 20 active consortia—teams of researchers, patients, and clinicians—each focused on a group of rare disorders. Join us for new episodes each month.Learn more about the RDCRN: www.rarediseasesnet... more

PublishesDailyEpisodes207Founded4 years ago
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Science

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Latest Episodes

New research from the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN). This summary is based on a paper published in the journal Molecular Genetics and Metabolism in March 2026 titled "A novel patient-Centered approach to clinical... more

New research from the Spastic Paraplegia Centers of Excellence Research Network (SP-CERN). This summary is based on a paper published in the journal Annals of Clinical and Translational Neurology on April 25, 2026, titled "Movement Disorders in Aicar... more

New research from the Urea Cycle Disorders Consortium (UCDC). This summary is based on a paper published in the Journal of Inherited Metabolic Disease on April 9, 2026, titled "Self-Reported Health-Related Quality of Life (HRQoL) in Adults With Urea ... more

New research from The IMPACT Study. This summary is based on a paper published in the journal Research and Practice in Thrombosis and Haemostasis on January 20, 2026, titled "Optimizing the management of congenital thrombotic thrombocytopenic purpura... more

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Frequently Asked Questions About Rare Research Report

What is Rare Research Report about and what kind of topics does it cover?

This program presents concise summaries of recent publications from a national network focused on rare diseases, with an emphasis on translating research into patient-centered care. Episodes commonly cover disease mechanisms, diagnostic advances, and emerging therapies across a broad spectrum of rare conditions, including leukodystrophies, urea cycle disorders, congenital thrombotic thrombocytopenic purpura, myasthenia gravis, and X-linked adrenoleukodystrophy. A recurring thread is the move toward early recognition, better outcome measurements, and more personalized management—ranging from clinical trial readiness and patient-centered endpoints to genome sequencing and biomarker approaches. The show often highlights the value of patient an... more

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Rare Research Report launched 4 years ago and published 207 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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