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

May 29th 2024

Patients with AATD had increased cardiovascular risk compared to non-AATD COPD and healthy controls, with results suggesting physiological tests may assess risk more accurately.

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

FDA Grants Nipocalimab Fast Track Designation for Rare Fetal Disease, with Katie Abouzahr, MD
FDA Grants Nipocalimab Fast Track Designation for Rare Fetal Disease, with Katie Abouzahr, MD

April 5th 2024

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

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