Advocacy

Leading advocates and experts share their experiences
Rare Disease Education via Art

Rare Disease Education via Art

Patricia Welton has two daughters with Ehlers-Danlos syndrome. Her daughters had the disease for years before properly being diagnosed.

Talking to Patient Groups is Good Business

Talking to Patient Groups is Good Business

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.

Mucopolysaccharidosis (MPS) Resource Center

Mucopolysaccharidosis (MPS) Resource Center

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).

RDLA Empowers Rare Disease Patients

RDLA Empowers Rare Disease Patients

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.”

The Rare Disease Film Festival

The Rare Disease Film Festival

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.

Defeating MSA (Multiple System Atrophy)

Defeating MSA (Multiple System Atrophy)

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.

  • Sign up
Lost your password? Please enter your username or email address. You will receive a link to create a new password via email.
We do not share your personal details with anyone.