Tag: Spinal Muscular Atrophy

Community News, News

Wythenshawe boy celebrates the birthday doctors thought he’d never see

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drug Spinraza, which can prolong and dramatically improve quality of life, was denied the youngster after funding was withdrawn by the NHS.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.

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Haris’ brother Maryus joins in the fun with Mickey and Mini Mouse

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.

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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.

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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.

Community News, News, Sport

Campaigning family continue to raise cash for baby Haris and disabled Wythenshawe kids

WhatsApp Image 2019-05-16 at 12.28.34 PMFundraising for baby Haris Khan is continuing with a raffle of a framed Manchester City away shirt, signed by the domestic treble-winning team.

There is also  Tyson Fury signed glove and a framed picture of the boxer signing the glove up for grabs.

Tickets are being sold in Wythenshawe Civic Centre until 8pm today (Monday) and the draw will take place outside Asda.

The family of Haris Khan, who suffers from Spinal Muscular Atrophy, won their battle to have the drug Spinraza, available on the NHS. The drug, which was previously available in Scotland and other countries but not England, will have a massive impact on the quality of life of Haris and other suffers across the country.

Dad, Shakeel, says the fund raising events are continuing  to buy a specially adapted vehicle for Haris and other disabled children in Wythenshawe,  with plans for days out for disabled youngsters in the area.

News, Politics

Campaigning family continues fight for”miracle” drug for baby Haris

A Wythenshawe dad of a terminally ill baby says time is running out for the NHS to provide a “miracle” drug which could massively improve the quality of his son’s life.

Eight-month-old Haris, from Newall Green, who has Spinal Muscular Atrophy was given three months to live when he was diagnosed with the condition in February.

Until February last year, the wonder drug Spinraza which can prolong and dramatically improve quality of life was available on the NHS, but was withdrawn on cost grounds.

Haris’ mum and dad Renata and Shakeel have been campaigning for the NHS restore funding for the drug, which is available in a number of countries including Scotland.

And in March the family and their supporters lobbied a meeting of the National Institute for Health and Care Excellence, the body which makes the recommendations for the funding of drugs on the NHS.

Following the meeting, NICE’s Chief Executive, Sir Andrew Dillon, wrote to Wythenshawe MP Mike Kane saying they were hopeful of  positive outcome with the drug company Biogen, who manufacture the drug.

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But nearly two months after their protest, there is still no decision on the funding of the drug which is deemed too expensive.

With Haris is now back home with his family in Newall Green, Shakeel says the NHS, NICE and Biogen all share responsibility for the failure to provide the drug with will have a massive impact on his son’s quality of life.

Funding for the drug will be considered again at meeting of NICE next week (Wednesday May 8) and NICE have released this statement:

Following the committee meeting on the 6 of March 2019, the appraisal committee asked NHS England and Biogen to enter into commercial discussions to see whether a managed access arrangement could be agreed for Spinraza consistent with NICE’s framework for determining cost effectiveness.

We have been informed by the parties that no agreement has yet been reached. Nevertheless, we consider it necessary to return to the appraisal committee next week in case an agreement is reached, or if no agreement is reached, for the committee to consider the consequences for guidance development.

This topic will be considered by the committee on Wednesday 8 May. Given the nature of the discussion, this will be held in a private.

Shakeel  says the fight will continue. Boxer Amir Khan is supporting the campaign and Shakeel says he has received tremendous support from the local community and from the charity New Life which has funded a special cot costing £4,000 and a buggy costing £3170. Haris also has a Just Giving page to raise funds in the event that the final decision is to reject funding the drug and the family has to go abroad to get treatment.

Spinal muscular atrophy is caused by dysfunctional or missing gene that affects the production of a protein essential for motor neurons. This condition is manifested by a shrinking spinal cord.

One in 40 people are a carrier of the faulty gene and in the most severe cases, such as Haris, children rarely live beyond two years, making SMA the leading cause of death in babies and toddlers in the country.

But Spinraza has been known to have significant effects on the length and quality of life for patients.

Manfuacturer’s Biogen markets Spinraza at $750,000 dollars in the USA during the first year and $375,000 per subsequent year. The company offered the drug at £450,000 to the NHS and an undisclosed discount, but it still was not enough.

One report has said Biogen’s growth is heavily dependent upon SMA medication.