- Title: “Development of a Novel Gene Therapy for Pompe Disease: Engineered Acid Alpha-Glucosidase Transgene for Improved Expression and Muscle Targeting”
- Session Date and Time:
Tuesday, Apr 30, 2019, 5:00 PM - 6:00 PM ET
- Session title: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases
- Room: Columbia Hall
- Abstract number: 518
These represent the first set of preclinical results for the Pompe gene therapy which Amicus is currently developing in collaboration with the Gene Therapy Program of the
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease. The disease can be debilitating, and is characterized by severe muscle weakness that worsens over time. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle. It is estimated that Pompe disease affects approximately 5,000 to 10,000 people worldwide.
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Vice President, Investor Relations & Corporate Communications
Director, Corporate Communications
Source: Amicus Therapeutics, Inc.