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.
Lysosomal Diseases NZ is a charitable trust dedicated to improve contact between families affected by lysosomal diseases within New Zealand, and supporting research into the causes and treatment of lysosomal diseases and improvements in the clinical care of affected people. Read more about our goals and activities.
Campaign for access to therapies for our diseases
We have been campaigning for over 10 years 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 escalated the issue to take a complaint to the Office of the Ombudsman, an officer of Parliament charged with scrutiny of the way government agencies administer policy. We are also working with political parties to achieve better systems for decisions about specialised medicines.
- 4th October 2013 - LDNZ, NZORD, MDA request Pharmac to reconsider declined applications for Freda and Yakuta
- September 2013 - LDNZ joins NZORD in seeking legal advice to Pharmac on Mandatory considerations