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 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…
- A summary of recent developments – 3 August 2018
- Meeting the Minister of Health Today – 3 April 2018
- The Health minister to the rescue? – 21 February 2018