by Peter Ciszewski | Jan 23, 2018
Julian Adams discusses Gamida Cell’s CordIn for rare genetic diseases. Each year, approximately 100,000 patients in the United States are living with sickle cell disease (SCD), a disease for which bone marrow transplantation is currently the only clinically...
by Peter Ciszewski | Jan 22, 2018
Sam Falsetti, PhD of Cambridge Biomarketing discusses some recent health care trends, specifically focusing on possible changes to the Affordable Care Act and drug development as it affects the rare disease community. Before private insurance market rules that were...
by Peter Ciszewski | Jan 22, 2018
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 | Jan 22, 2018
Annette Bakker, PhD, President and Chief Scientific Officer, of the Children’s Tumor Foundation (CTF) discusses her Foundation’s partnership with SpringWorks Therapeutics. SpingWorks is working with CTF on treatments for Neurofibromatosis.
by Peter Ciszewski | Jan 22, 2018
Len Walt, Vice President, Head of Medical Affairs, Sobi in North America, discusses his company’s clinical trial testing anakinra in Still’s disease, a rare inflammatory disorder. Still’s disease, a disorder featuring inflammation, is characterized...
