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Team
Our Approach
Innovative Therapies
Overview
X-Linked Myotubular Myopathy
Pompe Disease
Duchenne Muscular Dystrophy
Myotonic Dystrophy Type 1
Expanded Access Policy
Careers
News
Contact
Patients + Families
Team
Our Approach
Innovative Therapies
Overview
X-Linked Myotubular Myopathy
Pompe Disease
Duchenne Muscular Dystrophy
Myotonic Dystrophy Type 1
Expanded Access Policy
Careers
News
Contact
Patients + Families