SAVE OUR CHILDREN WE NEED NUSINERSEN NOW!
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Type 1 SMA is the most severe, leaving baby's like me with very complex complications, we are unable to sit,walk,crawl, feed, even breath and hold our own heads by ourselves.
Most babys dont make it to their second birthday and some lucky to see their 1st.
A common cold can easily turn into pneumonia, this usually takes the lives of these babies, along with respiratory failure.
We are three of four children in Northern Ireland with SMA type 1.
Although children with SMA Type 1 have limited movement, their brains are completely unaffected and they communicate through their big beautiful eyes and beaming smiles. They are very alert and seem to make up for their physical disabilities with their facial expressions, and they love to play just like any other child.
There are many different variations of SMA,
Type I – the most severe type. Symptoms appear in babies less than six months old, and they never develop the ability to sit unsupported.
Type II – less severe than type I. Symptoms usually appear in babies aged 7 to 18 months. Children with Type II can sit unsupported and some are able to stand, but they can't walk independently.
Type III – the mildest type affecting children. Symptoms usually appear after 18 months of age, and children are usually able to reach all the major motor milestones, including independent walking.
Type IV – affects adults.
There was no form of treatment for SMA until 23rd December 2016, when the American food and drug agency (FDA) announced that it had given full approval for the first treatment for SMA the drug spinraza/nusinersen, available to both children and adults.
We are awaiting approval from European Medicines Agency (EMA) for a licence for the drug in Europe.
The national institute for clinical excellence (NICE) is currently conducting what is known as a "scoping" process. This means it is gathering evidence about SMA and nusinersen should the EMA grant a licence.
Aside from this there is extended access programmes available across the uk for children with type 1 to be given the drug. An extended access programme is designed to provide access to treatment for eligible infants with SMA TYPE 1 who didn't participate in a clinical trial. The programme is aimed at providing access prior to any European Medicines Agency approval.
An EAP has opened in Belfast for those living in NI as the number of infants currently eligible for treatment is so small. One child in RBHSC has already received several doses of this drug and they do not plan on taking any more children on at this stage. Now this is hardly fair considering there is 3 other children eligible for the treatment which I am one of who have not be giving access to this potentially life saving and life changing drug. Not to mention any type 2 children who are very weak and would border on being type 1 which there is a few of in NI. This is vital medical treatment, Vital medical treatment that our kids are now being denied The drug is free as part of the extended access programmen. The cost of the administration and assessment of the drug will be covered by the NHS and local hospitals.
Our children have gone through enough and it is time that we all stood up and help them receive this VITAL treatment that they not only need but they deserve. Time is so precious SMA doesn't wait for no one. It is sadly to late for far to many baby. Its time we done something! OUR CHILDREN NEED EQUALITY AND THEY NEED IT NOW.
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