Clinical Insights

FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia

FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocytopenia

The CheckRare Brief delivers a concise summary of the week’s most important developments in rare diseases. Each episode covers FDA approvals, clinical trial updates, conference highlights, scientific publications, and industry news that matter to healthcare professionals, researchers, advocates, and the rare disease community.

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Medical Treatment and Stem-Cell Transplantation in Patients With Cutaneous T-Cell Lymphoma

Medical Treatment and Stem-Cell Transplantation in Patients With Cutaneous T-Cell Lymphoma

Lauren Shea, MD, discusses aspects of medical treatment and stem-cell transplantation in patients with cutaneous T-cell lymphoma (CTCL).

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🎙️ The first episode of our new podcast, The CheckRare Brief, is out now. Join us every week to learn about the latest in rare diseases and what this means for the rare disease community.

🎧 Watch episode 1 at the link below, or wherever you get your podcasts.

🎙️ The first episode of our new podcast, The CheckRare Brief, is out now. Join us every week to learn about the latest in rare diseases and what this means for the rare disease community.

🎧 Watch episode 1 at the link in our bio or wherever you get your podcasts.

...#CheckRare #TheCheckRareBrief #RareDiseaseNews

What is Itvisma gene therapy for patients with SMA?

John Day, MD, PhD, discusses its development and approval at https://checkrare.com/itvisma-gene-therapy-for-patients-with-spinal-muscular-atrophy/

#CheckRare #SMA #SpinalMuscularAtrophy #RareNeurology #RareMusculoskeletal ...#RareDiseaseTreatment

What is Itvisma gene therapy for patients with SMA?

John Day, MD, PhD, discusses its development and approval at https://checkrare.com/itvisma-gene-therapy-for-patients-with-spinal-muscular-atrophy/

#CheckRare #SMA #SpinalMuscularAtrophy #RareNeurology #RareMusculoskeletal ...#RareDiseaseTreatment

Initiation of FORZETTO Clinical Trial Testing Zeleciment Rostudirsen in Duchenne Muscular Dystrophy

🔎New Learning Center- Prader-Willi Syndrome

Learn more at https://checkrare.com/prader-willi-syndrome-underlying-causes-natural-history-and-management/

#CheckRare #RareDisease #PraderWilliSyndrome #RareGenetic

🔎New Learning Center- Prader-Willi Syndrome

Learn more at https://checkrare.com/prader-willi-syndrome-underlying-causes-natural-history-and-management/

#CheckRare #RareDisease #PraderWilliSyndrome #RareGenetic

Episode 1: FDA Advisory Board’s Concerns About New Duchenne Drug, Brain Fog in Immune Thrombocyto...