Maryam and her father Shakil Malji

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Shakil Malji

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Shakil Malji began a petition to get funding for his baby daughter Maryam’s drug treatment

A father who lost his son to a rare genetic disorder has won funding for his nine-month-old daughter’s “wonder drug” treatment for the same condition.

Shakil Malji’s daughter Maryam has type 1 spinal muscular atrophy (SMA1) which affects eating and breathing.

Her diagnosis came after access to the disorder’s only approved medication stopped, but a petition by Mr Malji received more than 350,000 signatures.

NHS England has now negotiated a deal to provide the treatment.

It agreed the deal with US manufacturer Biogen for the drug Spinraza, initially for a limited time to allow for more long-term data to be collected.

Nice said the treatment will be made available to the youngest and most severely-affected patients immediately, according to the Local Democracy Reporting Service.

At the time of Maryam’s diagnosis in December her parents were told she could have less than a year to live, but the NHS drug approval body Nice was not recommending Spinraza because of the “extremely high” costs.

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Shakil Malji

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Maryam’s brother died of the condition she has in December 2015

Mr Malji, from Newham, east London, 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 had been one of the first children to receive Spinraza on a medical trial but died aged one in December 2015 from an infection.

However, the overall trial was successful and led to the treatment being funded by governments in 24 European countries, including Scotland.

A Nice spokesman said: “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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