Amicus Therapeutics Honors Fabry Disease Awareness Month and International Pompe Day
April 17 2018 - 7:30AM
Amicus Therapeutics (Nasdaq:FOLD) is participating in several
activities in April to honor Fabry Disease Awareness Month and
International Pompe Day. The Fabry community has designated the
month of April to honor and recognize those who living with the
disease and to promote greater awareness. The goal of International
Pompe Day on April 15 was to foster global awareness of Pompe
disease.
In recognition of Fabry and Pompe awareness,
Amicus is participating in the following:
- United Pompe Foundation’s (UPF) 7th Annual Late-Onset Pompe
Disease Patient Meeting, hosted by the Duke Pompe Disease Clinical
and Research Program, Durham, NC, April 6-7
- Annual Fabulous Fabry Females Meeting, Emory University,
Atlanta, GA, April 22
- New Fabry disease educational websites launched by Amicus:
- FabryFacts.com: a global educational resource
providing information and resources about Fabry disease to support
healthcare professionals in diagnosing and caring for people with
Fabry disease. More information is available at
www.fabryfacts.com.
- FabryConnect: an interactive website for
people living with Fabry disease to learn, share, and connect with
the Fabry community. More information is available at
www.fabryfacts.com/fabryconnect.
"In the Amicus spirit of friendship, we join the
Fabry and Pompe disease communities in supporting several
activities to drive awareness during the month of April,” said John
F. Crowley, Chairman and Chief Executive Officer of Amicus
Therapeutics, Inc. “Each day at Amicus, through our research and
development efforts as well as our broader, employee-driven Healing
Beyond Disease initiatives, we seek to bring innovation and hope to
all people living with rare metabolic diseases."
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 the enzyme acid alpha-glucosidase (GAA). Reduced or
absent levels of GAA leads to accumulation of glycogen in cells,
which is believed to result in the clinical manifestations of Pompe
disease. Pompe 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 Healing Beyond
DiseaseHealing Beyond Disease is a cross-functional
initiative that was created and implemented by the employees of
Amicus Therapeutics to enhance and expand the company’s positive
effect on people living with rare diseases. Launched in 2018,
Healing Beyond Disease comprises elements of volunteerism,
mentorship, philanthropic giving, innovation and accessibility -
each inspired by and adaptive to rare disease communities. For more
information please visit the Amicus corporate website.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-centric
biotechnology company focused on discovering, developing and
delivering novel high-quality medicines for people living with rare
metabolic diseases. The cornerstone of the Amicus portfolio is
migalastat, an oral precision medicine for people living with Fabry
disease who have amenable genetic mutations. Migalastat is
currently approved under the trade name Galafold™ in the European
Union, with additional approvals granted and pending in several
geographies. The lead biologics program in the Amicus pipeline is
ATB200/AT2221, a novel, late-stage, potential best-in-class
treatment paradigm for Pompe disease. The Company is committed to
advancing and expanding a robust pipeline of cutting-edge, first-
or best-in-class medicines for rare metabolic 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, 2017. 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.
CONTACTS:
Investors/Media:Amicus
TherapeuticsSara Pellegrino, IRCVice President, Investor Relations
& Corporate Communicationsspellegrino@amicusrx.com (609)
662-5044
Media:Pure CommunicationsJennifer Paganelli
jpaganelli@purecommunications.com (347) 658-8290
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