Top Secret Charity Song Boasts massive line-up

10 September 2015

The All Blacks gathered yesterday to get a sneak first peek at a charity song featuring the creme of the Kiwi music industry crop which will launch tomorrow night. Cure Kids are raising funds to support the work of Prof David Palmer and his team at Lincolin university and to help save the lives of children affected by Batten disease. Read more.

Pharmac plea for more money rejected by Governement

18 August 2015

The Government has rejected Pharmac’s bid for more money in this year’s budget, despite the drug purchaser’s concerns it could not invest in new medicines. Read more here.

27th Feb 2015 – Pharmac in Negotiations over Rare Disorder Medicine

27 February 2015

Pharmac today released a press release advising they are still in negotiations with several drug companies as a result of its competitive process for rare disorders medicines. This announcement comes on the eve of Interantional Rare Disease Day. Read more here.

Abeona Therapeutics announce $3.6 million raised to develop treatment for Sanflippo Disease

8 December 2014

Cleveland, OH, December 1, 2014 – Biotechnology startup Abeona Therapeutics announced it raised $3.6 million to complete preclinical development of therapies for children with Sanfilippo syndrome (mucopolysaccharidosis type III, MPS II. Read more (link no longer active).

Genzyme’s Expression of Hope – Stage III

14 October 2014

Genzyme has opened stage III of their Expression of Hope Art (link no longer active) competition. Do we have any budding artists here in New Zealand who want to enter. Sarah Noble ML III won the featured artwork in 2009. Can we do it again?

August 2014 – NZORD Election Manifesto

25 August 2014

Over the weekend NZORD released the responses received from all political parties on NZORD’s election manifesto (link no longer active). It certainly makes for an interesting read. It is very disappointing but not unexpected that National has chosen to sit on the fence and again hide behind Pharmac in its repose to orphan drug access. United First is also fence sitting and stating they would support some policies on an evidence based response to any need for action. Having said that it is very pleasing to know that our voices are being heard.

April 2014 – High cost Medicines for Rare Disorders

9 April 2014

Phamac release their discussion document about possible access to high cost treatments. Is this a move to shut us down and not make this an election issue?

Pharmac’s Decision Criteria – Proposal for change

26 February 2014

Last year Pharmac held public meetings to consult on their decision criteria these links will take you to a Summary of Submissions and this link will take you to their proposal for change

FDA approves Vimizim to treat Morquio A

19 February 2014

FDA approves Vimizim to treat Morquio A. Read more. (link no longer active)

A Call to Action to all Lysosomal Families

19 July 2013

In our latest newsletter said that we would be getting back to you with a request to make submissions to Pharmac on their intent to decline funding for the drug Soliris for patients with PNH http://www.pnhsanz.org.nz/

I also indicated that should Pharmac get good support from the public to decline this treatment it would effectively shut the door to all rare disease therapies including our Lysosomal enzymes. We cannot let that happen.

Attached is my submission to Pharmac with some key messages. I am encouraging you all to make a submission using the attachment or take extracts out of it and put it into your own words. We want to flood Pharmac with submissions that tell them clearly that we do not support their intent to decline Soliris.

To read more about this proposal from Pharmac http://www.pharmac.health.nz/news/item/proposal-to-decline-a-funding-application-for-eculizumab

Submissions close on Wednesday 31st July and must be sent to

Email: eculizumabfeedback@pharmac.govt.nz