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 program update for Pompe disease
- Sarepta Therapeutics signs agreement with Lacerta Therapeutics, gaining rights to CNS-targeted Gene Therapy programs
- 2018 International Pompe Day #DYK
- Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society
- AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease
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