Drug Development

Jan 18, 2022| Posted in: Cancers, Drug Development, Treatment

Albumin-Bound Sirolimus: Newly-Approved Treatment for PEComa

Brendan Delaney, Chief Operating Officer of Aadi Bioscience,…
Jan 17, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Lung Diseases, News

MT1621: A Possible Treatment for Thymidine Kinase 2 Deficiency

Dr. Bradley Galer, MD, Chief Medical Officer  at…
Jan 13, 2022| Posted in: Cancers, Diagnosis, Drug Development, Hematologic Disorders, Treatment

What Physicians Can Learn About CML Treatment from the OPTIC Trial 

Dr. Thihan Padukkavidana of Takeda Oncology discusses what…
Jan 11, 2022| Posted in: Drug Development, Hematologic Disorders, News, Treatment

Positive Results from PRINCE Study of Pegcetacoplan in PNH Patients

Dr Monica Fay of Apellis Pharmaceuticals discusses results…
Jan 10, 2022| Posted in: Congenital And Genetic Conditions, Drug Development, Hematologic Disorders, News

Gene Editing Therapy Shows Promise for Sickle Cell Anemia 

Philippe Duchateau, PhD of Cellectis discusses the company’s…
Jan 6, 2022| Posted in: Cancers, Diagnosis, Drug Development, Hematologic Disorders

Disease-Free Survival vs. Progression-Free Survival in Cancer Studies

Dr Paolo Ghia discusses the difference between disease-free…

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.

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.

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.

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).

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: 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.

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