Latest Summaries

WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages. However, due to the heterogeneous presentation of the disease, coupled with lack of awareness of the condition, the diagnosis may be delayed, sometimes even into adulthood.

Tenosynovial Giant Cell Tumor (TGCT)

Tenosynovial giant cell tumor (TGCT) is a non-malignant tumor involving the joint synovium, bursae, and tendon sheath. These rare tumors are sometimes referred to as giant cell tumor of the tendon sheath (GCT-TS) and/or pigmented villonodular synovitis (PVNS).

CME Program: Gaucher Disease Highlights from WORLDSymposium 2020

Neal Weinreb, MD, FACP highlights the latest information about Gaucher disease from WORLDSymposium and provides expert analysis of its clinical relevance to help health care providers better care for patients with this rare disease.

CME Program: Fabry Disease Highlights from WORLDSymposium 2020

Ozlem Goker-Alpan, MD highlights the latest Fabry disease information from the WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with this rare disease.

Latest Summaries

Rare Genetic Disorders and Quality of Life Issues

I Stay Home For Rare

Why Brigatinib Was Approved to Treat ALK+ Non-small Cell Lung Cancer

What Dose of Ponatinib Is Both Safe and Effective to Treat CML?

Diagnosing Polycythemia Vera

Targeted Therapy for Acute Myeloid Leukemia

Isolation Due to COVID-19 May Create Another Problem

FDA Approves Drug to Treat Malaria

Protalix’ Fabry Disease Clinical Program

Treatment of AML in the Elderly Has Changed Significantly

FDA Approves New First-line Therapy for Rare Lung Cancer

PTG-300 Showing Promise to Control Hemocrit Levels in Polycythemia Vera

Duchenne Muscular Dystrophy

Data from Von-Hippel-Lindau Clinical Trial Shows Promise for HIF-2a Inhibitor

Overview of the Mucopolysaccharidoses

Can Boosting the Immune Response Help Treat COVID-19?

FDA Approves New Treatment for 4th-Line Treatment for GIST

FDA Approves New Drug for Kaposi Sarcoma

Glioblastoma

Fabry Disease Research Highlights: Earn CME/CNE Credit

WHIM Syndrome Learning Center

Tenosynovial Giant Cell Tumor (TGCT)

CME Program: Gaucher Disease Highlights from WORLDSymposium 2020

CME Program: Fabry Disease Highlights from WORLDSymposium 2020

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