Sickle Cell 101 conducts patient engagement interactions to better understand patient experiences in the sickle cell community.
Rare Disease International (RDI) is a global alliance of rare disease patient organizations designed to be an international voice for the rare disease community. One of its key initiatives is to include rare diseases in the Universal Health Coverage being developed by the United Nations (UN) for the year 2030.
Karolyn Lewandowski is the Chief Operating Officer & General Counsel of the Appendix Cancer Pseudomyxoma Peritonei (ACPMP) Research Foundation, a non-profit organization focused on sponsoring research towards pseudomyxoma peritonei, appendix cancer, and related peritoneal surface malignancies.
Jeffrey W Olin, DO, Professor of Medicine at the Icahn School of Medicine at Mount Sinai, discusses the Fibromuscular Dysplasia Society of America (FMDSA) and it’s FMD Registry.
Ilan Ganot, Co-Founder, President and CEO of Solid Biosciences discusses how his company views advocates and patients who have Duchenne muscular dystrophy (DMD). Mr. Ganot started Solid in 2013 to find treatments, and potentially a cure, for DMD, a disease that afflicts his son Eytani.
Kristen Wheeden, Director of Development for the American Porphyria Foundation, provides an overview of her foundation and it’s focus on physician education. The American Porphyria Foundation is also dedicated to the health and well-being of patient and families impacted by Porphyria.