Nieman-Pick Disease

by | Oct 9, 2017

Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses Niemann–Pick diseases, a subgroup of lipid storage disorders called sphingolipidoses in which harmful quantities of fatty...

Raising Awareness of Myeloproliferative Neoplasms (MPNs)

by | Oct 6, 2017

Emmy-Award Winning Actress Finola Hughes discusses why she is raising awareness for a group of rare, chronic and under-recognized blood cancers called Myeloproliferative Neoplasms (MPNs) Finola Hughes is dedicated to increasing awareness for rare blood cancers, while...

The Orphan Drug Act and Rare Disease Drug Pricing

by | Sep 29, 2017

Robert Derham, Founder of CheckOrphan discusses the Orphan Drug Act and some of the misconceptions surrounding it. Robert also talks about rare disease drug pricing. CheckOrphan: The Longest Running Website Focused Exclusively on Rare Diseases.

Diagnosing Myeloproliferative Neoplasms (MPNs)

by | Sep 28, 2017

Ellen Ritchie, MD, Associate Professor of Clinical Medicine, Weill Cornell Medical College, discusses the challenges of diagnosing Myeloproliferative Neoplasms (MPNs). MPNs are rare, chronic blood cancers. Chronic conditions last for a long period of time and may...