News Video | Policy | GPs | Hospitals | Medical | Mental Health | Welfare | Search

 

Family hopeful of receiving rare disease drug

Family hopeful of receiving rare disease drug available all over the world

Elizabeth Hunt’s family in New Zealand may finally get treatment for their rare genetic condition – treatment available to other family members who live in Australia, and around 30 other countries.

Elizabeth’s family has Fabry disease, a rare genetic disorder that leads to a shortage of an essential enzyme, which then causes damage to the heart, lungs, kidneys, skin, stomach and brain.

Two of her sons live in Australia and are eligible for Enzyme Replacement Therapy (ERT), while Elizabeth’s other son in Auckland is unable to access this life-changing medication.

“The disparity between countries means that my two sons in Australia have access to treatment, while my son James, like other Kiwis, does not,” says Elizabeth.

Now Elizabeth is hopeful that PHARMAC will finally fund suitable treatment for the estimated 35 Fabry patients in New Zealand. ERT has been available in Australia since 2004, and can also be accessed by patients in the UK and around 30 other countries.

PHARMAC is inviting pharmaceutical suppliers to submit funding applications for medicines for rare disorders. Applications close on Monday 3 September 2018 and will be considered by the Subcommittee in November.

“There are more than 7,000 rare disorders impacting over 377,000 New Zealanders all over the country,” says Chief Executive of New Zealand Organisation for Rare Disorders (NZORD) Dr Collette Bromhead.



“New Zealand is falling behind Australia and many other countries on treatment for these rare conditions, so we are very pleased that PHARMAC has initiated this invitation for medicines.”

Elizabeth has set up a Fabry Support Group to connect with other New Zealand families with the same condition https://www.facebook.com/Fabry-Support-Group-New-Zealand-158149027643895/

About NZORD

NZORD is an umbrella group set up to provide information, support and advocacy for patients and families affected by rare disorders, and to assist with the development of support groups for them. Rare diseases affect about 8% of the entire population.

Ends.


© Scoop Media

 
 
 
Culture Headlines | Health Headlines | Education Headlines

 

Joseph Cederwall Review: NZSO Plays Bach And Ellington

The NZSO’s Shed Series returned for 2019 with the aptly titled REBIRTH at Shed 6 on Friday 8th. This theme dealt with the way in which music constantly evolves and changes, with styles and musical periods reborn in completely new and different ways. More>>

Howard Davis Review: Voulez-Vous Couchez Avec Moi? - C'est Chic, Ça Colette!

Starring the ravishingly vibrant Keira Knightley in the title role, Colette is a fairly pedestrian paint-by numbers biopic - which is a little disappointing, given that its cross-dressing and pansexual protagonist lead anything but a dull life. More>>

Wellington Repertory Theatre: New Season Includes Royal Hunt of The Sun

“The objective behind our ambitious 2019 season is to expand our offering to a broader range of Wellingtonians. It’s the first time in over a decade that we’ve produced four shows, and the first time we will be performing at the Hannah Payhouse,” says Oliver Mander, WRT’s President. More>>


Howard Davis Review: The Revenants - They Shall Not Grow Old

Jackson has toiled laboriously in the trenches of the digital revolution for decades, with his two turgid Tolkien trilogies constantly pushing the envelope in terms of computer-generated entertainment and providing his latest project with sufficient combat experience to warrant serious Oscar consideration for its remarkable technical achievements. More>>


Unseen Let It Be Footage: Peter Jackson Making "The Ultimate Beatles Film"

Jackson's new film will be compiled from fifty-five hours of never-released studio footage of The Beatles recording their final album Let It Be in January 1969. More>>


NZCT Grants More Than $150,000 to Community Sports

NZCT has approved grants to the Tauranga Boxing Academy, provided a new lease of life to old yachts, benefitted sports at Tiniroto School, and helped to upgrade Christchurch City BMX Club track. More>>

 
 
 
 
 

LATEST HEADLINES

  • CULTURE
  • HEALTH
  • EDUCATION
 
 
  • Wellington
  • Christchurch
  • Auckland