Toddler would be left to die under New Zealand Health System
By Allyson Locke
My daughter Molly has an inherited disorder called Pompe Disease – a rare form of Muscular Dystrophy. Although there is
no cure for this disease, individuals that suffer from Pompe are given hope to live a relatively normal life with the
drug named Myozyme.
The reality is that 3 countries in the world, ONE being New Zealand, don’t fund this medicine due to the ‘cost.’
Molly (two years old) lives in Texas; USA and born to a New Zealand Mum, was diagnosed back in April of this year
through genetic testing and began enzyme replacement therapy(ERT) in June 2017. Regular blood work has shown that her
body is responding positively to the medicine that she receives through infusion every fortnight. The medicine has
proven to slow down the progression of the disease and Molly and other individuals with Pompe will be able to be
productive members of society – but not in New Zealand. Due to the political decisions in New Zealand, Molly and her
family can not move home and can’t even visit their family. In New Zealand Molly will be left with NO chance of
receiving Myozyme and will be left to die.
Very few parties have made a commitment to help save these people, but there has been positive communication from Labour
and New Zealand First which are determined to give Pompe sufferers a chance at life. As a New Zealander, I was lead to
believe New Zealand was the best place to live; we had opportunities, a government that actually cared about their
citizens, but parties such as National, have chosen to ignore funding this drug, ultimately denying those with Pompe a
chance at life. A change needs to happen, with your support Molly can come home.
Pompe Disease is an inherited disorder caused by a build up of glycogen in the body’s cells due to Molly not producing a
specific enzyme (acid alpha-glucosidase) that breaks down the complex sugar (glycogen) The accumulation of glycogen
affects certain organs, tissues, especially muscles. Molly currently experiences muscle weakness and mobility issues
along with reduced lung function and requires a wheelchair for her to keep up with her friends. As disheartening as it
was to hear that she has a fatal disease, I was given hope when I was told there was a treatment available called
Myozyme.
Myozyme isn’t a cure but it is treatment that has shown to help many Pompe sufferers around the world. We recently
returned from a conference held at Duke University in North Carolina. We met many patients that have Pompe, many of whom
discovered they had Pompe through new born screening. These individuals started their infusions as early as 3 days after
they were born and are now amazing individuals who keep up with their friends and maintain relatively normal lives.
Without the early detection and this amazing drug, I wouldn’t have met them at the conference simply because they
wouldn’t have made it to their first birthday.
As much as I enjoy living in America, my hopes were that my family would return to my home country and raise our two
kids the ‘kiwi way’ surrounded by their grandparents. I have recently discovered this will not be possible as Molly will
not be able to get her life saving drug in New Zealand. As a result, her health would rapidly decline and I choose not
to think what her future would be. I was once lead to believe that New Zealand was the best place to live, we had
opportunities, government officials that actually cared about their citizens- we are small town New Zealand. This
obviously isn’t the case when the government DENIES individuals the RIGHT to a medicine that has proven its worth all
around the world and is giving individuals a life, a life they deserve just like everyone else.
Molly does have extensive muscle issues and lung problems but after going to visit Dr Kishnani at Duke University, I am
so hopeful. It is giving her a life. It's truly heart breaking that it isn't offered in New Zealand.
I get asked often by my friends and family when I'm coming home - I've been away for over ten years! I can't come home
and sure as hell don't have my own mum in a time when I just need a hug and to be told it's going to be ok and that I'm
doing good.
One question I asked the fellow Pompe parents while I was at the conference this year, was how do you work, raise a
family, and get your child ERT every other week or weekly and still try to maintain a normal life? Their responses upset
me. They said they rely on the help and support of family and most don't work. I could have cried right then and there.
Oh how I wish I could have my parents to help me! I miss my parents, I miss my friends but my immediate family is my
number one priority. If Molly can't get her medicine, then I stay here and give her the quality of care she deserves and
I put my big girl panties on and try and forget about my feelings about a life in New Zealand.
New Zealand, you are hurting your image but more importantly, your people.
ENDS