AdvocacyLeading advocates and experts share their experiences
The Rare Disease Film Festival focuses exclusively on rare diseases will take place November 9-10, 2019 at the Mission Bay Conference Center in San Francisco.
Luke Rosen is the father of a daughter with KIF1A associated neurological disorder (KAND) and co-founder of the short-lived, but highly successful patient organization, KIF1A.org.
Neena Nizar, PhD, co-founder of the Jansen’s Foundation is the personification of the determination.
Patricia Welton has two daughters with Ehlers-Danlos syndrome. Her daughters had the disease for years before properly being diagnosed.
At Ovid Therapeutics, the patients’ input is essential to how they develop medications and how they do business. Leading that mindset is Luke Rosen, Patient Engagement & Digital Health Experience Head for the company.
Mucopolysaccharidoses (MPSs) are a group of genetic lysosomal disorders in which persons have low levels of specific enzymes that leads to an abnormal accumulation of complex carbohydrates (mucopolysaccharides or glycosaminoglycans).
Luke Rosen, father of a daughter with KIF1A associated neurological disorder (KAND), and co-founder of the KIF1A.ORG talks about the signs and symptoms of KAND and how it is the “perfect storm’ of systems that has led him and other parents to start a advocacy group to...
Christina Hartman, Senior Director of Advocacy & Policy for the EveryLife Foundation describes RDLA’s philosophy as “We don’t speak for the patients, but we empower the patients to have the knowledge and the tools to speak for themselves, and to advocate for these policies.”
Daniel DeFabio discusses the Rare Disease Film Festival, a relatively new event showcasing films from around the world which address the challenges of life with a rare disease. The first event was held in Oct of 2017 in Boston, MA.
Philip Fortier, executive director of Defeat SMA USA, Defeat SMA Canada, and the MSA Awareness Shoe. Multiple System Atrophy (MSA) is a rare, neurodegenerative disorder that largely affects the autonomic nervous system.