Amicus Therapeutics Honors Fabry Disease Awareness Month and International Pompe Day
In recognition of Fabry and Pompe awareness, Amicus will participate in the following:
- United Pompe Foundation's (UPF) Late-Onset Pompe Disease Patient Meeting, hosted by the Duke Pompe Disease Clinical and Research Program,
Durham, NC (April 7-9). - Fabulous Fabry Female Meeting,
Emory University ,Atlanta (April 9). - Company-wide participation in #PauseforPompe awareness campaign (April 15).
- 2017
Fabry Support & Information Group (FSIG)Fabry Expert Conference ,Cincinnati, OH (April 28-30).
"Fabry Disease Awareness Month and International
About Fabry Disease
Fabry disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including
pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to organ damage regardless of the time of symptom onset.
About Pompe Disease
Pompe disease is an inherited lysosomal storage disorder caused by deficiency of an enzyme called acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to the accumulation of the substrate glycogen in the lysosomes of muscles and other tissues. Progressive accumulation of glycogen is believed to lead to the morbidity and mortality associated with Pompe disease, including muscle weakness and respiratory insufficiency.
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Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any
or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended
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CONTACTS: Investors/Media:Source:Amicus Therapeutics Sara Pellegrino Senior Director, Investor Relations spellegrino@amicusrx.com (609) 662-5044 Media: MWW PRSid Dinsay sdinsay@mww.com (646) 381-9017
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