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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Genetic Variants in STAT4 Linked to Disabling Pansclerotic Morphea: JAK Inhibition Shows Promising Results
Genetic Variants in STAT4 Linked to Disabling Pansclerotic Morphea: JAK Inhibition Shows Promising Results

June 2nd 2023

Gain-of-function STAT4 variants in patients with disabling pansclerotic morphea suggest a potential therapeutic role for JAK inhibition, resulting in improved clinical symptoms and inflammatory markers.

Robert Hariri, MD: Natural Killer Cell Therapy Shows Remarkable Potential in Treating Relapsed AML
Robert Hariri, MD: Natural Killer Cell Therapy Shows Remarkable Potential in Treating Relapsed AML

May 31st 2023

Ziritaxestat Fails to Demonstrate Efficacy for Treatment of IPF in 2 Trials
Ziritaxestat Fails to Demonstrate Efficacy for Treatment of IPF in 2 Trials

May 16th 2023

FDA Approves Pegunigalsidase Alfa for Adults with Fabry Disease
FDA Approves Pegunigalsidase Alfa for Adults with Fabry Disease

May 10th 2023

Nintedanib Remains Safe for SSc-ILD Through 3 Years
Nintedanib Remains Safe for SSc-ILD Through 3 Years

May 9th 2023

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