Latest Summaries

Aug 3, 2020| Autoimmune / Autoinflammatory Disorders, Cancers, Lung Diseases, News

COVID-19 and Lung Cancer Patients

Ross Camidge, MD, PhD of the University of…
Jul 30, 2020| Cancers, Hematologic Disorders, Treatment

Typical Treatment Plan for Polycythemia Vera Patients

Ronald Hoffman, MD, Director of the Myeloproliferative Diseases…
Jul 25, 2020| Cancers, Hematologic Disorders, News, Regulations, Treatment

FDA Approves CAR T Therapy for Mantle Cell Lymphoma

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

Lymphoma Research Foundation Overview

Meghan Gutierrez, chief executive officer of the Lymphoma…
Jul 17, 2020| Cancers, Drug Development, Hematologic Disorders, Treatment

Follicular Lymphoma: Diagnosis, Prognosis, and Treatment

John Leonard, MD of Weill Cornell Medicine discusses…
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…

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.

Rare Disease Clinical Trials CME Program

Learn about clinical trials and rare diseases through these brief courses that are certified for CME and CNE credit.
  • Heather Lau, MD, MS, NYU School of Medicine, New York, NY
  • Morie A Gertz, MD, Mayo Clinic, Rochester, MN
  • Timothy Craig, DO, Penn State University, Hershey, PA
  • Eric T. Wong, MD, Harvard Medical School, Boston, MA

CME Program: Mucopolysaccharidoses (MPSs) Highlights from WORLDSymposium 2020

Barbara K. Burton, MD highlights the latest information about MPSs from WORLDSymposium and provides expert analysis of its clinical relevance for members of the care team in order to help them care for patients with these rare diseases.

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.

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.

CME Program: Managing Individuals With Eosinophilic Granulomatosis with Polyangiitis (EGPA)

Michael E. Wechsler, MD, MMSc provides an overview of Eosinophilic Granulomatosis with Polyangiitis (EGPA) – including how to shorten individuals’ diagnostic journey and the time till they access effective therapy – through these brief courses that are certified for CME credit.

TTR Amyloidosis CME Program

Morie A Gertz, MD, MACP provides an overview of TTR Amyloidosis – including how to shorten individuals’ diagnostic journey and the time till they access treatment which might improve their quality of life or even prevent disease progression – through these brief courses that are certified for CME credit.

Spinal Muscular Atrophy CME Program

Nancy L. Kuntz, MD, FAAN discusses diagnosing and managing people with spinal muscular atrophy through these brief courses that are certified for CME and CNEcredit.

Disease Categories

Topics

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.

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.

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.

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.

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).

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.

  • Sign up
Lost your password? Please enter your username or email address. You will receive a link to create a new password via email.
We do not share your personal details with anyone.