Complex and infinitely challenging, finding diagnoses for rare diseases takes real heroes that lead the charge in this battle. These unsung heroes aren’t looking for glory or for recognition. What they need is for us to help them promote their journey in the charge against rare diseases so that together, we can have a bigger impact and make a greater difference!
- Audentes Therapeutics’ “Be Bold for Courageous Patients” Program to Support Patients Living with Rare Diseases
- Amicus Therapeutics establishes a 75,000 sq. ft. Gene Therapy Center of Excellence
- Amicus Therapeutics Receives Breakthrough Therapy Designation for Pompe Treatment
- Rare Disease Day 2019
- Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
advocacy awards Awareness Breakthrough therapy designation CAP chaperone clinical trials compassion CORD eLearning Emma Rooney ERT Extraordinary Measures Fabry fundraising Gaucher gene therapy getting treated grants guest blogger Int'l Pompe Day IPA late-onset living with Pompe meetings Muscular Dystrophy Canada MyNormal newborn screening Orphan Drug Framework Orphanet politics pre-clinical preclinical pregnancy Rare Disease Rare Disease Day research respite video