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!
- Sarepta Therapeutics Enters into a Strategic Relationship with Aldevron in Support of Their Gene Therapy Program
- CAP Walks for Muscular Dystrophy in 2018
- Amicus Therapeutics Announces First Patient Dosed in Phase 3 PROPEL Study of AT-GAA in Patients with Pompe Disease
- Audentes Therapeutics Pompe Program Update
- Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies
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