CRANBURY, N.J., April 06, 2017 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of rare and orphan diseases, is supporting several activities during Fabry Disease Awareness Month and International Pompe Day. The Fabry community has designated the month of April to honor and recognize those who are affected by the disease and to promote greater awareness. The goal of the Fourth International Pompe Day on April 15 is to foster global awareness of Pompe disease.
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 Pompe Day are excellent opportunities for us to unite with the Fabry and Pompe communities to elevate disease awareness, in particular the significant unmet needs for people living with both diseases,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “Our patient-centric vision at Amicus is to drive awareness and continuous innovation to deliver significant benefits to people living with these rare genetic diseases for many years to come.”
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.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.
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 December 31, 2016. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
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Tagged: Int’l Pompe Day