Latest Summaries

Jul 9, 2020| Advocacy, Diagnosis, Drug Development, Regulations, Treatment

NCATS and Drug Development’s “Valley of Death”

Donald Lo, PhD, Director of the National Center…
Jul 7, 2020| Cancers, Hematologic Disorders, News, Treatment

Accelerated Approval of Tazemetostat for Follicular Lymphoma

John Leonard, MD of Weill Cornell Medicine discusses…
Jun 29, 2020| Cancers, Hematologic Disorders, Treatment

Combination of Venetoclax and Azacitidine To Treat Acute Myeloid Leukemia

Courtney Dinardo, MD from MD Anderson Cancer Center…
Jun 25, 2020| Congenital And Genetic Conditions, Kidney And Urinary Diseases

What is Primary Hyperoxaluria Type 1 (PH1)?

Jaap Groothoff, MD of the University of Amsterdam…
Jun 24, 2020| Cancers, Hematologic Disorders, Treatment

Venetoclax-Obinutuzumab vs Chlorambucil-Obinutuzumab To Treat CLL

Othman Al-Sawaf, MD of the University Hospital of…
Jun 16, 2020| Cancers, Endocrine Disorders, Metabolic Disorders

Acromegaly

Acromegaly is a rare endocrine disorder due to…

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.

Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the central nervous systems of young patients.

Accelerating Time to Diagnosis: The Global Commission to End the Diagnostic Odyssey for Children With a Rare Disease

The Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease is a multidisciplinary collaboration of experts from around the world co-chaired by Takeda, Microsoft and EURORDIS-Rare Diseases Europe, who have brought together their creativity, technological expertise and commitment to accelerate the time to diagnosis for children with a rare disease.

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.

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.

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