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.

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

June 18th 2024

Long-term Phase 2 data demonstrate KP104's potential as an optimal first-line monotherapy for PNH.

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

FDA Issues CRL to Abeona for Potential RDEB Cell Therapy
FDA Issues CRL to Abeona for Potential RDEB Cell Therapy

April 23rd 2024

Video Interviews
Podcasts
Autoimmune Hepatitis at ACG 2023, with Gina Choi, MD

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