What are lysosomal storage diseases?
Lysosomal storage diseases are inherited genetic defects that result in an enzyme deficiency, which prevents cells from performing their natural recycling function. This leads to a variety of progressive physical and/or mental deterioration. Approximately 180 New Zealand families are currently affected. Find out more.
Our online petition
Campaign for access to therapies for our diseases
We have been campaigning hard since the early 2000s to get access to specialised therapies for our diseases. Some progress has been achieved but there is still a long way to go. We have made numerous submissions, organised seminars, and taken delegations to officials, politicians and to Pharmac itself. Our campaign included a complaint to the Office of the Ombudsman, an officer of Parliament charged with scrutiny of the way government agencies administer policy, plus investigations of possible legal action. We are also working with political parties to achieve better systems for decisions about specialised medicines. Read more...
- 30th November 2016 - Enzyme Replacement therapies - 3 new decisions and one more review
- 8th November 2016 - Rare Disease Mum Abandoned by her local MP