For all those that are still reading my blogs, updates and general moans I have a sensitive one coming up and I’d really love for your help and support. A friend of mine has a seriously lovely friend who in 2014 gave birth to a gorgeous baby boy – Maddox.
All went well through her pregnancy, scans and birth. The day they were discharged from hospital was the day the world came crashing around a little bit; it became apparent in the first few days of Maddox’s life that although he was perfect, he in fact wasn’t a healthy baby as had been predicted.
After many tests and consultant visits Maddox was diagnosed with HPP (or to give it its ‘proper’ name Hypophosphatasia). Now you may not have heard of this – and why would you have? – it’s an ultra rare bone disease that affects only 7 people a year in England.
HPP is a life-shortening disease which affects bone mineralisation and a major symptom of this is respiratory distress. Perinatal and infantile HPP are the worst and mortality rates in the first year are as high as 97%.
However, Maddox is thriving as well as he can be with a child of his condition and is getting ready to enjoy his second Christmas with his Mummy, Daddy and new baby sister.
So why is Maddox in the 3% of survivors?
This is because from 2.5 weeks old; Maddox and Mummy spent 3.5 months at the Birmingham Children’s Hospital where he received specialist treatment and trialled a new drug called Streniq or Asfotase Alfa. This is the only treatment for babies and children with HPP. Quite simply, this is a lifesaving drug. Since he was three weeks old Maddox has received this via subcutaneous injections which his mum and dad still continue to do for him now, three times a week.
A recent evaluation of this “miracle drug” by NICE has recommended that the NHS should no longer provide this life saving treatment. This recommendation makes the future for Maddox and others diagnosed with this life-limiting disease uncertain and challenging.
But together we can overturn this decision!
Understandably there are cost implications, as there is with all treatment, however I firmly believe if sufferers can be given the chance then they should. I would hate to think that the proposed stop of this drug is down to its (estimated) £367K price tag!
This amount is a drop in the ocean for the price of a family watching their little boy grow up and reach milestones that were deemed impossible without Streniq/Asfotase Alfa. And who has the right to put a price on a child’s life?
Most of you have a couple of minutes to spare in your waking day. So, if there’s one good thing you do today – make it this:
1. Register here: https://www.nice.org.uk/guidance/indevelopment/gid-hypophosphatasiaasfotasealfaid758/consultation/html-content
2. Post a comment with a short story/reason of why this drug simply cannot be stopped.
3. Share this status.
This means so much to this family and little Maddox so please, please take the time to Register, Comment and Share.
Thank you x