Shire, a global biotechnology company, enables people with life-altering conditions to lead better lives by developing innovative, specialty medicines to meet significant unmet patient needs.  Since 2011, Shire has been providing its enzyme replacement therapy (ERT), VPRIV® (velaglucerase alfa for injection), on a humanitarian basis to children in Egypt with Gaucher Disease through a Project HOPE program in that country. 

Gaucher Disease is a rare, inherited metabolic disorder.  The debilitating symptoms of Gaucher Disease are caused by a deficiency of a crucial enzyme, glucocerebrocidase.  Shire’s VPRIV is designed to replace that enzyme and is indicated for long-term use for patients with Type I Gaucher Disease.  While treatment for Gaucher Disease is readily available in the developed world, there is a critical shortage of access to treatment in lesser-developed nations.

Reda Mansour, M.D. (left) with a patient

Project HOPE identified a group of young patients in Egypt who were in dire need of treatment.  Without humanitarian assistance, these patients had a poor prognosis.  According to Reda Mansour, M.D, Chief of Party for Project HOPE in Egypt, “Most of the patients in this program were in critical condition, with a poor chance of survival without therapy. Now they are smiling and continuing to make progress.”

All of the patients benefiting from Shire’s program are closely monitored by local specialists at five children’s hospitals in Egypt. Additional medical oversight is provided by an independent expert medical committee comprised of internationally renowned Gaucher Disease specialists.

Beyond providing much needed medicine free of charge, Shire is also supporting Project HOPE’s work in Egypt by increasing public awareness of Gaucher Disease and training local clinicians in proper management of the disease through financial contributions.

“We at Shire are very pleased to help make possible such a meaningful impact on the lives of these patients and their families,” said Hartmann Wellhoefer, M.D., Head of Medical Affairs, Rare Diseases, at Shire.


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