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Shedding light on an ultra-rare blood disease

Shedding light on an ultra-rare blood disease

Daniel Webby to address public seminar on World Rare Disease Day today – Thursday, February 28, 2013

Auckland-based artist, Daniel, 32 has one life-long wish –to pursue an ordinary life with his six-year-old son, Wu.

Recently diagnosed with an ultra-rare, life-threatening blood disease that claims the lives of one-in-three-patients within five years of diagnosis, if untreated, Daniel will today reveal his personal story of living with Paroxysmal Nocturnal Haemoglobinuria (PNH) at a public seminar marking World Rare Disease Day (February, 28, 2013).

Hosted by the New Zealand Organisation for Rare Disorders (NZORD) at the Rydges Hotel, Wellington, this morning’s seminar will feature a host of presenters with an interest in, or living with, a rare disease. The seminar will examine access to specialised medicines for rare “orphan” diseases.

World Rare Disease Day is an annual awareness-raising event designed to improve access to treatment and medical representation for those living with rare diseases and their families.

According to Daniel, “There are approximately 7,000 known rare diseases worldwide, thousands of which are prevalent in New Zealand today.

“World Rare Disease Day offers our PNH community, as one of the 147-strong patient support groups comprising NZORD, an opportunity to generate public awareness of our ultra-rare disease, known to affect less than 20 Kiwis.

“It also presents us with a platform to call on PHARMAC to urgently subsidise the only effective treatment of its kind for our disease, for those whose lives so desperately depend upon it,” said Daniel.

PNH is a silent killer that affects people in the prime of their lives. On average, the disease strikes people in their mid-30s, however 10 per cent of all people with PNH start developing symptoms before they’re 21. The median survival for people with PNH is 10 years post-diagnosis.

“PNH patients simply don’t have time up their sleeves. The disease can trigger a range of health complications including fatigue, poor physical function, fatal blood clots, chronic kidney disease, anaemia, stroke, heart failure and vital organ damage,” Daniel said.

“Ultimately the constant destruction of red blood cells overwhelms the body, and the waste material cannot be processed, resulting in clots developing in almost any organ.”

Complications relating to the disease almost claimed Daniel’s life last year. He has since endured a slow and painful road to recovery.

“Because PNH is an ultra-rare disease, it’s difficult to show or tell people what it’s about, or what’s going on, because it all happens internally. It can be incredibly isolating,” said Daniel.

“The situation is really grave. PNH is deadly and members of our community are growing sicker by the day, battling a progressive disease that claims lives.

“The only effective treatment for PNH, Soliris, is cost-prohibitive without Government subsidy,” Daniel said.

“Members of our small patient association could benefit significantly from PHARMAC granting us subsidised access to this treatment.

To learn more about Daniel’s situation and the plight of other Kiwis living with PNH, and their fight for access to effective subsidised treatment, attend this morning’s public seminar in Wellington, or tee up an interview with Daniel.

About the PNHSANZ
The PNHSANZ was established by Daniel and other concerned New Zealanders in 2012 to fight for access to the only effective treatment available for PNH. The association is one of 147 patient support groups comprising NZORD. For more information about PNH, or to sign their ‘Petition for life’, visit

About PNH
PNH is an ultra-rare, progressive and life-threatening disease known to affect less than 20 New Zealanders. The disease is characterised by ‘haemoloysis’ or the destruction of red blood cells, which can cause a variety of major health problems such as the formation of blood clots, high blood pressure and damage or failure of organs including the brain, liver, gastro-intestinal system and kidneys.

Various symptoms, including abdominal pain, difficulty swallowing, poor physical function, shortness of breath, and debilitating fatigue, can also compromise a patient’s quality of life.

Estimates suggest one-third of patients with PNH do not survive more than five years from the time of diagnosis.

About World Rare Disease Day
Rare Disease Day was founded in Europe on February 29, 2008. With the original intention of celebrating the day every four years (leap years), momentum and enthusiasm for the day spurred an annual celebration, with events now being staged world-wide. New Zealand began participating in World Rare Disease Day in February 2010.

Formed in September 2000 following a conference of more than 30 rare disease support groups, the NZORD network has since grown to 147 support groups. The organisation aims to provide New Zealanders affected by rare disorders and their families with resources and information. NZORD also monitors rare disease issues and policy matters, while continuously building partnerships between patients and their families, support groups, clinicians, researchers, policy-makers and industry.


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