Latest Summaries

Jun 4, 2020| Advocacy, News

I Stay Home For Rare

Levi Gershkowitz, founder of #Istayhomeforrare explains the origins…
Jun 4, 2020| Cancers, Lung Diseases, Treatment

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

Ross Camidge, MD, PhD of the University of…
Jun 3, 2020| Cancers, Hematologic Disorders, Treatment

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

Jorge Cortes, MD from the Georgia Cancer Center at…
Jun 2, 2020| Cancers, Hematologic Disorders, Treatment

Diagnosing Polycythemia Vera

Ronald Hoffman, MD, Director of the Myeloproliferative Diseases…
Jun 1, 2020| Cancers, Hematologic Disorders, Treatment

Targeted Therapy for Acute Myeloid Leukemia

Eunice Wang, MD, Chief of the Leukemia Service…
May 29, 2020| Autoimmune / Autoinflammatory Disorders

Isolation Due to COVID-19 May Create Another Problem

Gary Grohmann, PhD, Director of the Australian Immunisation…
May 28, 2020| News, Regulations, Treatment

FDA Approves Drug to Treat Malaria

The  U.S. Food and Drug Administration (FDA) has…
May 27, 2020| Congenital And Genetic Conditions, Drug Development, Lysosomal Storage Disorders

Protalix’ Fabry Disease Clinical Program

Dror Bashan, President and CEO of Protalix Biotherapeutics…
May 26, 2020| Cancers, Hematologic Disorders, Treatment

Treatment of AML in the Elderly Has Changed Significantly

Courtney  DiNardo, MD, Associate Professor at the University…
May 25, 2020| Cancers, News, Regulations, Treatment

FDA Approves New First-line Therapy for Rare Lung Cancer

The U.S. Food and Drug Administration (FDA) has…
May 22, 2020| Cancers, Hematologic Disorders, Treatment

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

Ronald Hoffman, MD at The Icahn School of Medicine…
May 21, 2020| Musculoskeletal Diseases

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive form of…
May 21, 2020| Cancers, Congenital And Genetic Conditions, News, Treatment

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

Eric Jonasch, MD, Professor, Department of Genitourinary Medical…
May 20, 2020| Congenital And Genetic Conditions, Metabolic Disorders, Musculoskeletal Diseases

Overview of the Mucopolysaccharidoses

Heather A. Lau, MD, Director, Lysosomal Storage Disease…
May 20, 2020| Autoimmune / Autoinflammatory Disorders, Drug Development, News, Treatment

Can Boosting the Immune Response Help Treat COVID-19?

Dr. Gary Grohmann, Director of the Australian Immunisation…
May 18, 2020| Cancers, News, Regulations, Skin Conditions, Treatment

FDA Approves New Drug for Kaposi Sarcoma

The U.S. Food and Drug Administration (FDA) has…
May 15, 2020| Cancers

Glioblastoma

Glioblastoma is a very aggressive brain tumor. Most…

WHIM Syndrome Learning Center

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.

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