August 2018 newsletter

1 October 2018

Here is the link to our very latest newsletter.

Rare Disease Epidemiology – 2nd Edition

20 February 2018

In mid-2017, John was invited to write a preface for a book called Rare Diseases Epidemiology, which concentrates on the work done for LDNZ and ISMRD and talks about what patient advocates can achieve.

Here’s the link to the PDF or you can pay for and download the book here.

Performance Report

18 December 2017

LDNZ Performance Report for 2017 can be found here.

April 2017 newsletter

2 May 2017

Here is the link to our very latest newsletter.

Pharma Company gives up on Pharmac

After many years of advocacy and Genzyme trying to work with Pharmac to fund Myozyme for our NZ patients with Pompe disease, the company has finally given up and is compassionately funding four Pompe patients. Read our press release.

13th December 2016 Petition Delivered to Government

19 December 2016

Samantha Lenik and patients with Pompe and Fabry disease and their supporters delivered the petition to the government yesterday requesting that the government look under urgency to ensure Pharmac has enough funds to properly deliver on the rare disease drug policy. Read more here.

Petition presentation rescheduled to Tuesday 13th December 2016

30 November 2016

Our plans for a 15 November event were disrupted by the significant earthquake the day before in Wellington. Now we are set to go again on Tuesday 13 December at 12.30pm. We will gather at the Seddon Memorial. Will you join us?

Remember, any signed petition sheets should be returned to Jenny Noble by Friday 9 December so the numbers can be tallied up in time.

ERT – 3 new decisions and one more review

There is some very positive news to report this month, and some disappointment as well. LDNZ is delighted that Pharmac has approved the listing of Myozyme for infantile Pompe disease on the schedule. This is the first approval for this treatment and putting it on the schedule means that any baby diagnosed with this disease, and who fits the treatment guidelines, will be automatically given treatment, avoiding delays and anxious waits for special consideration under the exceptional circumstances scheme. Read more.

PTAC reviewing Myozyme for adult Pompe disease

2 November 2016

At its November meeting, PTAC, which is Pharmac’s technical advisory committee, will be reviewing the evidence of effectiveness for Myozyme for adult Pompe disease. This is happening in parallel with the consultation on listing of three enzyme replacement therapies on the schedule of funded treatments. These are exciting times for us. There is some progress, but keep gathering signatures on the paper petition. We need to keep the momentum going. Remember to get the signed sheets back to Jenny by Friday 11 November, so they can be tallied up and prepared for the presentation at Parliament on Tuesday 15 November.

Our Petition for Medicine Access 2016

23 September 2016

LDNZ is calling on all Lysosomal families, friends and supporters to actively support our petition to the House of Representatives and to Health Minister Jonathan Coleman, to ensure that Pharmac has enough funds to implement its rare disease policy.

  • You can sign our on-line petition here¬†or
  • You can download our paper petition to gather signatures from friends and family here