Rare in Common: The Challenges of Rare Disease Families

by | Jan 23, 2018

Alisa Shakarian of Cambridge Biomarketing discusses the film Rare in Common and the importance of telling indidivuals’ rare disease stories in a visual manner. Rare in Common follows the experiences of rare families: their struggles, their strength, and their...

Sickle Cell Disease and Gamida Cell’s New Therapy

by | 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...

Health Care Trends to Look for in 2018

by | 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...

Neonatal Onset Multisystem Inflammatory Disease (NOMID)

by | 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....