The Orphan Drug Act: What Does “Orphan” Mean?

Douglas Paul, PharmD, PhD discusses the Orphan Drug Act and what the term “Orphan” means in context of the the Rare Disease industry, especially considering that much of the Rare Disease pipeline is for Oncology products. The Orphan Drug Act was first...

Ovid Therapeutics: Focusing on Rare Neurologic Diseases

Jeremy Levin, Chairman and Chief Executive Officer of Ovid Therapeutics, discusses his company’s focus on developing medicines for patients with rare neurological disorders and how neurology compares to where oncology was 20 years ago. Ovid Therapeutics is a New...

Clinical Trial for X-Linked Retinoschisis

David G. Birch, PhD, Chief Scientific and Executive Officer and Director of the Rose-Silverthorne Retinal Degenerations Laboratory, Retina Foundation of the Southwest discusses the current clinical trial for patients suffering from X-linked retinoschisis (XLRS)....

Choroideremia Research Foundation

Cory MacDonald, Operations Manager, Choroideremia Research Foundation, discusses the origins of his organization and the various clinical trials for this rare eye disorder. Choroideremia is a condition characterized by progressive vision loss that mainly affects...