Amicus Therapeutics Honors Fabry Disease Awareness Month and International Pompe Day
April 06 2017 - 8:15AM
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 DiseaseFabry
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 DiseasePompe
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.
Forward-Looking StatementsThis
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.
FOLD–G
CONTACTS:
Investors/Media:
Amicus Therapeutics
Sara Pellegrino
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
MWW PR
Sid Dinsay
sdinsay@mww.com
(646) 381-9017
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