Dean Suhr, President of the MLD Foundation, provides an overview of Metachromatic leukodystrophy (MLD) and an overview of his advoacy organization.
Jennifer McNary is a consultant, speaker, and rare disease advocate. She is also a mother of two boys with Duchenne muscular dystrophy. In this video, Jennifer discusses Rare Disease Day and her evolving role of Advocate to Industry consultant.
Dr. Collins began his talk by acknowledging that Rare Diseases are more relevant that ever with all of NIH’s 27 institutes and centers having some investment in rare disease research.
Kyle Bryant is a rare disease advocate. In this video, Kyle discusses Friedreich’s Ataxia (the rare disease he lives with) and Rare Disease Day. Kyle was diagnosed with Friedreich’s Ataxia at the age of 17.
Rare Disease Day is a day for those in the rare disease community to share their stories. There are over 7000 rare diseases and each person who has a rare disease, or who is working to find a treatment for a rare disease, has a unique story to tell. CheckRare, …
Steve Silvestri, Director of Public Policy at the EveryLife Foundation for Rare Diseases, discusses the role of advocates in lobbying Congress and the 2019 Legislative Conference Agenda held February 25, 2019 at the Ronald Reagan Building and International Trade Center in Washington, DC.