Regulations
Urgency For Galactosemia Treatment
Dr. Shoshana Shendelman, President and CEO of Applied…FDA Approves Momelotinib To Treat Myelofibrosis
Momelotinib is a potent inhibitor of Janus kinase…What is Ben’s Friends
Ben Munoz, co-founder of Ben’s Friends, talks about…A Possible Breakthrough in Acromegaly Treatment
Acromegaly, a serious and rare disease caused by…Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center Designated as Rare Disease Center of Excellence by NORD
Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center…FDA Expands the Use of Luspatercept as First Line Therapy for Anemia In Patients with MDS
This approval expands on an earlier indication that…FDA Approves Pozelimab to Treat Ultra-rare Autoimmune Disorder (CHAPLE disease)
CHAPLE disease is a genetic autoimmune disorder that…FDA Approves Palovarotene to Treat Fibrodysplasia Ossificans Progressiva (FOP)
The cost of the ultra-rare drug will be…FDA Approves Elranatamab to Treat Relapsed/Refractory Multiple Myeloma
The orphan drug is approved for patients who…Phish Finder and the Undiagnosed Patient
Dr. Kim Moran, Head of US Rare Diseases…
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: 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.

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: 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.

Rare Disease Clinical Trials CME Program
- 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