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

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

Video Interviews
Podcasts
Latest CME Events & Activities

Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care

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Clinical ConsultationsTM: Focusing on Fabry Disease in Females: Individualizing Diagnostic and Counseling Plans to Improve Long-Term Outcomes

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Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making

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C.R.U. Eye Symposium (Current, Relevant, Useful)

11/10/2023-11/12/2023

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C.R.U. Eye Symposium (Current, Relevant, Useful)

11/10/2023-11/12/2023

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C.R.U. Eye Symposium (Current, Relevant, Useful)

11/10/2023-11/12/2023

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Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices

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Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices

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Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices

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Advances In™ Desmoid Tumors: Recognizing Disease Burden, Pathophysiology, and Targeted Treatments in Development

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BURST CME™: Taking ALS Management to the Next Level

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