The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.

Latest News

US FDA logo in black over a white background. |Credit: US Food and Drug Administration
Mirum Pharmaceuticals Submits NDA for Chenodiol Tablets in Cerebrotendinous Xanthomatosis

June 28th 2024

Mirum Pharmaceuticals submitted an NDA for chenodiol tablets to treat CTX based on positive phase 3 RESTORE trial results.

KP104 Achieves Robust Efficacy in Paroxysmal Nocturnal Hemoglobinuria | Image Credit: Fotolia
KP104 Achieves Robust Efficacy in Paroxysmal Nocturnal Hemoglobinuria

June 18th 2024

Elizabeth Sapey, MBBS, PhD | Credit: University of Birmingham
Traditional Assessment Tools May Underestimate Cardiovascular Risk in AATD

May 29th 2024

Zane Elfessi, PharmD | Credit: LinkedIn
A1AT Phenotyping Lags Behind, Contributes to Underrecognition of AATD

May 10th 2024

US FDA logo in black over a white background. | Credit: US Food and Drug Administration
FDA Approves Mavorixafor (XOLREMDI) as First Drug Indicated for WHIM Syndrome

April 29th 2024

Video Interviews
Podcasts
New Insight with Veeral Sheth, MD Podcast logo | Image Credit: HCPLive
Autoimmune Hepatitis at ACG 2023, with Gina Choi, MD

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