MPS Society Welcomes New Enzyme Replacement Therapy for Rare Genetic Disease - MPS II (Hunter syndrome)



    Elaprase (TM) offers hope for children and adults with MPS II (Hunter
    syndrome)

    VANCOUVER, June 28 /CNW Telbec/ - People with MPS II (Hunter syndrome)
have new hope now as Health Canada has approved ELAPRASE(TM), a human enzyme
replacement therapy, for sale in Canada, following priority review. Elaprase
is the first and only treatment approved for people suffering from MPS II, a
rare, life-threatening genetic condition that results from the absence or
insufficient levels of the lysosomal enzyme iduronate-2-sulfatase. Elaprase,
which is given as weekly infusions, replaces the missing enzyme that MPS II
patients fail to produce in sufficient quantities.
    MPS II is usually diagnosed in children aged 18-36 months. It is
progressive, and life-limiting. There is a broad range of severity in the
symptoms. Untreated, the disease may result in a thickening of heart valves,
an enlarged liver and spleen and other organs, obstructive airway disease, and
progressive deafness. It may also result in changes in facial features,
skeletal changes including joint stiffness, and in some cases central nervous
system involvement leading to potentially serious developmental delays. It is
estimated that approximately 40 people in Canada and 2,000 people worldwide
are afflicted with MPS II.
    "Making Elaprase available in Canada will mean a world of difference for
families across the country whose children are living with MPS II. This is a
progressive disease, so the sooner a child is treated, the better," said
Kirsten Harkins, Executive Director of The Canadian MPS Society. "It was only
in February that the first child in Canada with MPS II began receiving
Elaprase through Health Canada's Special Access Program and reimbursed by the
BC government. We know there are more families waiting for the treatment to be
made available so that their children can have a better quality of life.
Elaprase will not cure the disease, but it is shown to have a remarkable
impact on the course of this devastating illness. This is very exciting news,
because now when a child is diagnosed with MPS II, he or she has the hope of
leading a normal life," said Harkins.
    Trey Purcell, a three-year-old B.C. boy with MPS II, has been receiving
weekly infusions since Feb. 5, 2007, after the treatment was made available
through the Special Access Program and special funding from the provincial
government. "Elaprase has given us hope that Trey will live a long and
fulfilled life," said Deb Purcell, Trey's mother. "We are thrilled with the
changes we have seen in Trey in only four months. His organs are shrinking
back to size, his facial features are softening, he is growing like a weed
again, and these are only the changes we've seen from the outside. As a parent
of a child with a progressive and life threatening disease, knowing that
there's treatment and hope means everything."
    Simon Ibell, an MPS II patient who was part of the clinical trial for
Elaprase, has defeated the odds and, at 29, is enthusiastic about this new
treatment. "Elaprase is a blessing and a life saver that I never thought would
be available in my lifetime. It has provided many positive changes, such as
improvements in my breathing, stamina, mobility, flexibility and decrease in
the size of certain organs. All of these improvements have provided a better
quality of life, even for me at my age. Also, I have witnessed the increased
benefits of Elaprase for those younger Hunter syndrome patients and I hope to
see this treatment being reimbursed across Canada in order to treat the
disease before it has progressed."
    "Health Canada's approval is a very important step, but only the first
step in the process. It is urgent that people with MPS II have access to this
treatment within their provinces," said Harkins. "We ask the provinces to act
quickly and approve funding for this new breakthrough treatment."




For further information:

For further information: Kirsten Harkins, Executive Director, The
Canadian MPS Society, (604) 924-5130, kirsten@mpssociety.ca; To arrange
interviews with patients, spokespeople or geneticists: Judith Walker/Jeff
Domansky, Peak Communicators, jwalker@peakco.com/jdomansky@peakco.com, (604)
689-5559; Charles Pitts, Oromedia, (877) 990 9044, cpitts@oromedia.com

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THE CANADIAN MPS SOCIETY

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