Nieman-Pick Disease

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...

The Orphan Drug Act and Rare Disease Drug Pricing

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)

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...

The Link Between Lipds and the Origin of Myelomas

Dr. Pramod Mistry discusses the link between lipids and the origin of myelomas, a type of cancer affecting plasma cells. Multiple myeloma is a cancer involving the growth of plasma cells, which are immune cells that make antibodies to fight infection. Uncontrolled...

Misconceptions Around the Orphan Drug Act

Douglas Paul, PharmD, PhD discusses some of the misconceptions around the Orphan Drug Act and how these myths are doing a great disservice to the Rare Disease Industry, specifically the pharma and biotech companies who are not getting the proper credit for the many...

Understanding X-Linked Retinoschisis (XLRS)

David G. Birch, PhD, Chief Scientific and Executive Officer and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, Retina Foundation of the Southwest discusses X-Linked Retinoschisis (XLRS). X-linked retinoschisis (XLRS) is an inherited early onset...
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