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Forest Gate baby with rare genetic disorder to receive 'wonder drug'

PUBLISHED: 09:15 15 May 2019 | UPDATED: 12:25 15 May 2019

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

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

Archant

A baby from Forest Gate with a rare genetic condition is set to receive a 'wonder drug' that could potentially save her life.

Maryam with her father Shakil. Picture: Rachael BurfordMaryam with her father Shakil. Picture: Rachael Burford

Little Maryam Malji's parents Zainab and Shakil were told she could have just months to live after being diagnosed with type 1 spinal muscular atrophy (SMA1), a disorder which affects walking, eating, drinking and breathing.

Her brother Abdullah also had SMA1 and was placed on a medical trial for drug Spinraza.

The one-year-old died from an infection in December 2015 but the trial was successful and led to Spinraza being government-funded in 24 European countries - although not in England, where the NHS approval body NICE said it was rejecting it due to its cost.

Friends, relatives and supporters of the little girl campaigned to allow her to receive the drug, with West Ham MP Lyn Brown even raising the issue in parliament.

West Ham MP Lyn Brown has been campaigning to help little Maryam Malji. Picture: Office of Lyn BrownWest Ham MP Lyn Brown has been campaigning to help little Maryam Malji. Picture: Office of Lyn Brown

And now NHS England has agreed to fund the drug, also known as Nusinersen, after reaching an agreement with manufacturer Biogen.

It means the NHS will fund the drug for a limited period of time, allowing further data to be collected on its effectiveness, but will be available to the youngest and most severely affected patients - including Maryam - immediately.

It costs £75,000 per dose and is administered by four injections into the cerebral spinal fluid, followed by a top-up injection every four months.

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While not a cure, trials have shown that Spinraza can slow the effects of SMA in some cases, allowing babies and toddlers to develop stronger muscles and survive for longer without breathing support.

Meindert Boysen, director of the centre for health technology evaluation at NICE, said: "We are very pleased that we can now recommend Spinraza for people with SMA.

"The committee has recognised that Spinraza is a promising treatment that has been shown to improve a range of outcomes important to patients. But it also recognised that there are significant uncertainties, particularly around its long-term benefits.

"All along we have felt it important to give all parties every opportunity to try to find a way to mitigate these uncertainties in order to make Spinraza available to patients in England."

Ms Brown welcomed the news, saying: "Months of campaigning by parents, councillors, imams, bishops, and my fellow MPs has finally paid off.

"It has taken far too long, but beautiful nine-month-old Maryam and other babies will be able to access treatment for SMA on the NHS.

"I'm going to keep pushing to ensure that access is rolled out immediately to all the families who need it."

She added: "It's clear from our experience on this campaign that procedures for approving funding for devastating rare diseases like SMA aren't working well. We need rapid progress on Kuvan, Orkambi and more. For families to have to wait so long while their children's health deteriorates is simply cruel.

"I'm overjoyed that this decision has finally been taken, but there is still work to do."

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