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'It's such a relief': Dad's delight as baby set to receive 'wonder drug'

PUBLISHED: 13:21 16 May 2019

Maryam with her father Shakil. Picture: Rachael Burford

Maryam with her father Shakil. Picture: Rachael Burford

Archant

A Forest Gate father who lost his son to a rare genetic disorder said he "cannot describe his happiness" after the NHS agreed to fund a "wonder drug" for his baby daughter who also has the condition.

Maryam Malj has type 1 spinal muscular atrophy and may just have months to live. Picture: Rachael BurfordMaryam Malj has type 1 spinal muscular atrophy and may just have months to live. Picture: Rachael Burford

Shakil Malji's nine-month-old daughter Maryam has type 1 spinal muscular atrophy (SMA), which affects eating, drinking and breathing.

When she was diagnosed with the deadly muscle-wasting condition in December, doctors told her parents she could have less than a year to live.

The family were devastated to learn an access programme to the new drug Spinraza, the only approved medication for the disorder, had been stopped just weeks before Maryam's diagnosis and the NHS drug approval body Nice was not recommending the treatment due to its "extremely high" cost.

Mr Malji set up a petition, which received more than 350,000 signatures with help from religious leaders and MPs, to get the drug funded, and led rallies outside Nice's offices in London and Manchester with other campaigners.

NHS England will now provide the treatment after "negotiating a deal" with US manufacturer Biogen. It will initially fund treatment for a limited time, allowing more data to be collected on its long-term effectiveness.

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Mr Malji said: "I'm still in shock. It is such a relief. Maryam and all the other children who have this condition now have a chance at life. We have so many people to thank — locally, nationally and internationally — who helped with this campaign. When the news came I cannot describe how happy everyone was."

Maryam's brother Abdullah also had SMA1 and was one of the first children to receive Spinraza on a medical trial. He died aged one in December 2015 from an infection, but the overall trial was successful and led to the treatment being funded by governments in 24 European countries, including Scotland.

An access programme mainly paid for by Biogen was rolled out at Great Ormond Street Hospital in 2016 and showed extremely promising results. But it ended in October last year, leaving dozens of babies in England with the condition unable to receive the drug. At the time Spinraza cost £75,000 per dose. It is administered by four injections into the cerebral spinal fluid, with a top-up injection every four months.

Councillor Mas Patel, who campaigned for Maryam to get the drug, said: "I feel so privileged to have been part of this amazing campaign. From Maryams parents, to local residents and our wonderful MP, it's really been a team effort and now Maryam and countless babies like her with SMA, will finally get the treatment they so badly need. We will save lives."

West Ham MP Lyn Brown, who also helped the family's battle, added: "Months of campaigning by parents, councillors, imams, bishops and my fellow MPs has finally paid off … I'm overjoyed this decision has finally been taken."

The treatment will be made available to the youngest and most severely affected patients immediately, Nice said.

A spokesman added: "Where companies show appropriate flexibility, it is possible to find a way to provide important treatments to patients in a cost-effective way."

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