Gaucher Community News - National Gaucher Foundation

Advancing Treatment Through
Ongoing Research
Submitted by Actelion
Since 1997 Actelion’s founders, Drs. Martine
and Jean-Paul Clozel, have led the company with
a commitment to research and the well-being of
patients. In the fight against rare orphan diseases,
Actelion is committed to bringing innovative,
highly advanced medicines that can make an
important difference in people’s lives and address
unmet medical needs.
Most recently, our team has been busy analyzing
results of a clinical study, that looked at the
long-term safety and efficacy of a treatment for a
genetic disorder. The two-year study completed in
late 2010, and the results were recently presented
for review to an expert investigator meeting. The
results of the study will be presented publically at
an upcoming medical congress.
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A Team Approach to Supportive Care
While we continue to support research into orphan
diseases, we’re also focused on making certain
people who take Actelion’s therapies receive
great support. Actelion works closely with the
specialty pharmacies like CuraScript to provide
supportive care. Staffed by a caring team with
extensive experience in diverse clinical settings,
CuraScript has centralized resources for people
who are receiving treatment. The CuraScript
team provides a wide range of services that start
right from the first time they talk to a patient.
Benefit specialists help patients navigate through
the managed care maze and ensure that insurance
issues are being addressed. Nurses are there to
discuss therapy and answer questions about side
effects, dosing, and other concerns patients may
have. And pharmacy techs monitor the phones 24
hours a day and follow up with monthly contact.
CuraScript reports, “We understand people have
a lot going on, so having someone to talk to each
month helps keep them focused on doing the best
they can on their therapy
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