Nov 28, 2017
Amit Rakhit, MD, Chief Medical and Portfolio Officer of Ovid Therapeutics, discusses the importance of investigating Ovid’s therapies in younger patients suffering from Fragile X and Angelman syndromes. Fragile X syndrome is a genetic condition that causes a... Nov 28, 2017
Amit Rakhit, MD, Chief Medical and Portfolio Officer at Ovid Therapeutics, discusses OV101, a compound that was originally developed to treat insomnia, but is now being studed in Angelman and Fragile X syndromes. OV101 (gaboxadol) is believed to be the only delta... Nov 24, 2017
Melissa P. Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, New York City, discusses the challenges of recruiting patients for rare disease clinical trials, including Nieman-Pick disease. Nov 24, 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... Nov 23, 2017
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... Nov 23, 2017
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... 
