Seriously ill tot denied lifesaving drug her brother helped trial before he died

A devastated dad has told of his heartbreak after his seriously ill daughter was denied a lifesaving drug – despite her older brother having helped trial it.

Shakil Malji’s five-month-old daughter Maryam has been diagnosed with Type 1 Spinal Muscular Atrophy (SMA) – the same condition his son Abdullah had.

Before his tragic death from an infection, Abdullah’s parents allowed their son to trial a drug named Spinraza, and said they noticed a dramatic improvement in his health.

But despite it being available in Scotland after a successful trial, Spinraza has not been licensed in England because of its high cost.

The family, from Newham in East London, now face a race against time to get the drug to Maryam.

Doctors have warned she could have just months to live – and Mr Malji said drug company Biogen has a moral duty to help.

Mr Malji told Mirror Online: "It’s terrible that they’ve got something available that can treat a sick child and they’re not using it because of money.

"They used our son which I’m not happy about because now his sister needs the drug they’re not helping."

He said the drug could save Maryam’s life.

Mr Malji said: "Right now you can see that she’s got a lot of weakness, but as time goes on she’ll get weaker.

"The longer they leave it, her muscles will weaken more and more, and they will only be able to save what’s left. We need to stop the deterioration.

"It’s stressful to me that a sick child might die as a result."

Children with the condition do not tend to live beyond two years unless they get the right treatment, he said.

And he is tragically aware that without a quick resolution, Maryam faces a lot of pain as her condition deteriorates.

He said: "She won’t be distressed at this time, but she could get distressed in three or four months time."

The drug is available in 24 European countries after the trial Abdullah participated in before his death in December 2015, was successful.

What is Type 1 SMA?

Children with type 1 SMA show symptoms in the first 6 months of life.

Babies with the condition:

• have very weak and floppy arms and legs (hypotonia)
• have problems moving, eating, breathing, and swallowing
• are unable to raise their head or sit without support

Most babies with type 1 SMA die during the first few years of life, usually as a result of serious breathing difficulties.

SMA Support UK has more information about type 1 SMA.

Source: NHS

He was just 13 months old.

Mr Malji said: "We helped them with our previous child, but now his own sibling can’t benefit from it."

The case has been taken up by the family’s local MP, Labour’s Lyn Brown.

A petition calling for Maryam’s family to be given access to the drug has been signed more than 135,000 times.

Ms Brown said: "This is a really tragic case. The family has already lost one child to this awful disease, and that child took part in Biogen’s trials for this new drug, which is now being denied to his sister.

" I’ve written to Biogen, suggesting they have a moral duty to give the family the drug, to Secretary of State Matt Hancock, and to Great Ormond Street Hospital. I will be taking part in a debate in Parliament on Tuesday to raise this issue again. Biogen should do the decent thing by this family."

Mr Malji said he wants the law tightened up to ensure that patients who take part in drug trials are able to access the medication if it’s successful.

He also claimed this should be extended to family members.

The high cost of Spinraza, also known as nusinersen, meant the National Institute for Health and Care Excellence (NICE) has not recommended it for use in England.

Meindert Boysen, director of the centre for health technology evaluation at NICE, said last year: “The committee accepted that SMA is both rare and a very serious condition that can have a severe impact on people with it, as well as on family members and carers, and that there is an unmet need for effective treatments.

"The committee was willing to be flexible because of the nature of the condition and the paucity of the evidence, but the very high cost of nusinersen meant it could not recommend the drug as a cost effective use of NHS resources."

But discussions are ongoing between Biogen and NHS England.

In a statement Biogen told Mirror Online: "Biogen cannot comment on individual cases but can confirm it has responded directly to the family concerned.

"The NHS is responsible for treating people living with spinal muscular atrophy (SMA) in England, and we continue to collaborate with NICE and NHS England to work towards a positive outcome for patients.

"Biogen recognises that the delay in accessing Spinraza (nusinersen) is deeply frustrating and concerning for SMA patients and their families, and we share that concern.

"England is already significantly behind many parts of the world in making nusinersen available."

It said it remains in discussions with NICE and NHS England about the drug.

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