Aims of the Irish MPS Society
The aims of the Irish MPS Society are as follows:
- to act as a support network for those affected by MPS and related diseases
- to bring about more public awareness of MPS and related diseases
- to promote and support research into MPS and related diseases
Through the MPS Society, we offer those who are affected by the
MPS diseases both general and practical support, ranging from informal
and social contact through background information to practical steps
to improve the lifestyle of those affected by the disease - both
sufferers and their families.
Increasing Public Awareness
Many people's first knowledge that MPS syndromes exist
is when a child or relative is diagnosed with the condition. Almost
invariably, the first reaction to any mention of MPS or the MPS Society
receives the same reaction - "What's that?". Our
society hopes to change that, by increasing public awareness of
these rare genetic syndromes.
Promoting & Supporting Research
Enzyme replacement therapies are now available for the treatment of MPS I (Aldurazyme(R) (laronidase)) and for MPS VI Naglazyme(R) (galsulfase) wholly developed and commercialized by BioMarin.
Aldurazyme is manufactured by BioMarin and marketed by Genzyme Corporation.
BioMarin Pharmaceutical Inc. announced its program for its third enzyme replacement therapy (ERT) for the treatment of mucopolysaccharidosis IVA (MPS IVA), or Morquio A Syndrome.
BioMarin plans to initiate a Phase 1/2 clinical trial in the first quarter of 2009 and expects to report initial results in the first half of 2010.