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

£80m new drugs fund for Wales by end of year

1 August 2016

A new £80m fund to help patients in Wales with life-threatening illnesses get quicker access to innovative and new medicines has been confirmed. Read more…

Researchers find conceivable alternative way to treat Pompe disease

27 July 2016

Researchers at Duke Health have identified a potential new avenue for treating Pompe disease, a rare condition caused by the build-up of glycogen, a storage form of sugar, in cardiac and skeletal muscle, the liver and other tissues, due to deficiency of a particular enzyme. Read more.

Sanfilippo Research Fund

Help LDNZ support New Zealand research into Sanfilippo disease. Top quality research at Massey University points to effect treatments for patients by studying the disease in huntaway dogs. But there are significant costs to be met to breed, feed, house and care for the dogs between research grants. Help us meet our goal of $35,000 to support the dogs and research by donating on our Givealittle fundraiser page.

BioMarin Close BMN701 Pompe Trials

1 July 2016

LDNZ received notice on 22nd June that BioMarin intends to close down the BMN 701 trials and that the 22nd July will be the last infusion for our 3 New Zealand patients. This is devastating for our patients as it leaves them high and dry with no other option for therapy. Allyson Lock one of the patients on the BioMarin trial talks with John Campbell: see the interview.

Stem Cell Gene Therapy trail for Sanfilippo Disease

13 June 2016

Scientists in Manchester have developed a stem cell gene therapy to reverse a fatal childhood illness, and have agreed to work with a new therapeutics company to test it in a human trial for Sanfilippo disease.

Read more

Pharmac announce listing of Naglazyme

17 March 2016

It was 11.22 am on 17 March 2016 when LDNZ got the briefing about this – the first listing in New Zealand of a novel treatment for a lysosomal disease since 1999 when Cerezyme was listed for Gaucher disease. Read more.

LDNZ Submission to Pharmac on Naglazyme Consultation

2 March 2016

Pharmac’s third consultation under the orphan drugs fund is for Naglazyme, for the treatment of Maroteaux-Lamy syndrome (MPS6), which is one of our Lysosomal diseases. LDNZ submitted this response in February 2016 and we wait now for Pharmac’s decision. This might be the breakthrough we have waited for, for so long – the possible funding for the first time from this special fund for rare diseases, of a treatment not previously accessible at all by patients in New Zealand. Read our submission here.

Canadian researchers break blood-brain barrier with new ultrasound treatment

11 November 2015

Canadian scientists have made history with a world first, successfully using focused ultrasound to break through one of the human body’s final frontiers – the blood-brain barrier. Read more. [Link no longer active.]

Rare Disease Day 2016

26 September 2015

Theme:  Patient Voice

Slogan: Join us in making the voice of rare diseases heard.

2016 marks the ninth year that the international rare disease community celebrates Rare Disease Day. On 29 February 2016, people living with or affected by a rare disease, patient organisations, politicians, carers, medical professionals, researchers and industry will come together in solidarity to raise awareness of rare diseases. Read more.

LDNZ is planning a virtual morning tea for the lysosomal community watch our website for more information.