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...

The Limited Treatment Options for Rare Diseases

Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City discusses the limited treatment options for some of the rare diseases she is focused on, including Krabbe disease, Nieman-Pick disease, and...

Challenges of Diagnosing Rare Diseases

Amit Rakhit, MD, Chief Medical and Portfolio Officer of Ovid Therapeutics, discusses the challenges of diagnosing rare disorders in general, and Fragile X and Angelman syndromes specifically. Family physicians, pediatricians, and other primary health care...

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...
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