Amicus Therapeutics to Present Preclinical Pompe Disease Gene Therapy Data at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting
Poster Presentation:
- 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.
About
Forward-Looking Statements
This press release contains "forward- looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended
CONTACTS:
Investors/Media:
Vice President, Investor Relations & Corporate Communications
spellegrino@amicusrx.com
(609) 662-5044
Media:
Director, Corporate Communications
mwinkler@amicusrx.com
(609) 662-2798
FOLD–G
Source: Amicus Therapeutics, Inc.