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!
- Amicus Therapeutics Enters Research and Development Collaboration with University of Pennsylvania to Develop AAV Gene Therapies
- Spark Therapeutics Announces New Preclinical Data for Their Pompe Disease Gene Therapy Candidate
- Amicus Therapeutics Announces Positive 18-Month Data in Pompe Disease Phase 1/2 Study
- Audentes Therapeutics program update for Pompe disease
- Sarepta Therapeutics signs agreement with Lacerta Therapeutics, gaining rights to CNS-targeted Gene Therapy programs
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