Your ability to recruit patients - to identify the most qualified patients, to make enrollment as simple and seamless as possible, and to provide support from application to randomization - is the major determinant to...
In this episode, Aaron speaks with Laura and Cameryn, a mother and daughter duo, about a rare disease called spinocerebellar ataxia (SCA) and the way it's impacted their lives.
Researching a new therapy for an ultra-rare disease: A look into the science of Enzyvant with Dr. Alex Solyom, senior medical director
We were lucky enough to have Dr. Alex Solyom join us for the pod. Dr. Solyom is the senior medical director at Enzyvant, where he and the team research a new therapy for an ultra-rare disease called Farber disease.
A newborn screening is a routine procedure. Shannah didn't give it much thought as she brought her daughter, Emmalyn, home from the hospital. But a late-night call set her down a road towards advocacy that she couldn't have predicted.
In this episode, we speak with Peter Morley, a self-described "accidental advocate" who, despite living with multiple chronic conditions, fights on behalf of all Americans for fairer healthcare access.