by Peter Ciszewski | Nov 7, 2017
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....
by Peter Ciszewski | Nov 7, 2017
The Food and Drug Administration (FDA) granted regular approval to Alectinib (Genentech) for treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), as detected by an FDA-approved test. In December 2015,...
by Peter Ciszewski | Nov 6, 2017
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...
by Peter Ciszewski | Nov 6, 2017
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...
by Peter Ciszewski | Nov 4, 2017
Overview hATTR amyloidosis is an inherited, rapidly progressive, life-threatening disease.1-3 hATTR amyloidosis is caused by a mutation in the transthyretin (TTR) gene that results in misfolded TTR proteins accumulating as amyloid fibrils in multiple tissues in the...
