Dr. Wasserstein Discusses 3 Rare Diseases

Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses three rare diseases that she sees at her clinic: Neimann Pick Disease, Krabbe Disease, and Chromosome 22q11.2 deletion syndrome...

Still’s Disease Background

Len Walt, Vice President, Head of Medical Affairs, Sobi in North America, provides an overview of Still’s disease, a rare inflammatory disorder that affects the entire body, and how these patients typically present to their physicians. Still’s disease is...

Myths Surrounding the Orphan Drug Act

Douglas Paul, PharmD, PhD discusses some of the myths surrounding the Orphan Drug Act, especially that Pharma and Biotech companies are gaming the system. These myths, Dr. Paul believes, could have long-term negative effects on Rare Disease drug...

Diagnosing 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 how X-Linked Retinoschisis (XLRS) is diagnosed. X-linked retinoschisis (XLRS) is an...

Neonatal Onset Multisystem Inflammatory Disease (NOMID) Overview

Len Walt, Vice President, Head of Medical Affairs, Sobi in North America, discusses neonatal onset multisystem inflammatory disease (NOMID), a disorder that causes persistent inflammation and tissue damage primarily affecting the nervous system, skin, and joints....

Targeted Therapies for Neurology Patients

Jeremy Levin, Chairman and Chief Executive Officer of Ovid Therapeutics, discusses his company’s focus on developing targeted therapies for patients with rare neurological disorders. Ovid Therapeutics is a New York-based biopharmaceutical company using its...
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