It was the party doctors feared may never happen, but the family of Wythenshawe boy, Haris Khan celebrated his first birthday at the weekend.
Haris, from Newall Green, who has Spinal Muscular Atrophy was given three months to live when he was diagnosed with the condition in February.
And the “miracle” drug Spinraza, which can prolong and dramatically improve quality of life, was denied the youngster after funding was withdrawn by the NHS.
But following a successful campaign by Haris’ mum and dad Renata and Shakeel, the NHS restored funding for the drug, with dramatic results.
And the family had a party to remember to celebrate Haris’ milestone. Mickey and Mini Mouse dropped by to join in the fun and there was a message of support from former international footballer Jermaine Defoe.
Shakeel and Renata thanked local people for their support , Sarah Ryder who donated marquee, Be Our Guests who donated the sweet car, t Pieceofcake who donated cakes and Serenes homemade party bags.
Children throughout England will benefit from the drug, which was already available in Scotland.
The National Institute for Health and Clinical Excellence (NICE), manufacturers Biogen and NHS England say they have agreed to make Spinraza available for children, young people and adults with SMA Types 1, 2 and 3 through a scheme known as a Managed Access Agreement (MAA). This means that patients will be able to get Spinraza while more long-term data on its effectiveness is gathered.
NHS England says the treatment will be made available to the youngest and most severely-affected (SMA Type 1) patients immediately by Biogen, with NHS England offering funding on NICE’s publication of final guidance.
It says that for older babies, children and young adults with SMA Types 2 and 3, the NHS will begin to provide Spinraza shortly after NICE’s guidance is published, once the services to deliver them are established. This is not expected to take more than a few weeks.
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