PKU sufferers plead for wonder drug to be made available – ‘we want to eat normal food’ | UK | News (Reports)

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PKU campaigners thank Daily Express for support over Kuvan

PKU sufferers endure a severely restricted diet as too much protein causes brain damage, so they have to avoid meat, fish, eggs, dairy and some vegetables. The Daily Express is fighting for the NHS to end its 12-year delay and buy the drug Kuvan, which would enable sufferers to eat more normal meals. Now 11 youngsters with the cruel genetic condition have filled a box of “Festive Wishes” and sent it to Kuvan’s maker BioMarin.

They are asking the US firm to lower its prices so the NHS can buy it for the 600 sufferers who could benefit.

The box was delivered to BioMarin’s London headquarters in Holborn by PKU sufferer Holly Graham, 24, of Godalming, Surrey. The music student said: “It contained young children’s Christmas wishes.

“I really hope this year they come true.

“I have lived with PKU as a small child, an adolescent and now as an adult. I know what they go through at that young age.

PKU

A box containing children’s wishes was delivered to BioMarin’s London headquarters by Holly (Image: Jonathan Buckmaster)

“I really hope these beautiful little children do not have to live without Kuvan as I have had to.”

The 11 brave sufferers who wrote wishes to BioMarin were Maria-Mi Dogaro, aged three; Rory Thompson, five; Florence Love, five; Jayden Infante Woodhall, eight; Oliver Hubble, one; Olivia Brady, one; Willow Hodder, two; Reo Fairbrother, 10; Stanley Brown, six; Saibah Hussain, one and Zain Mohammed Iqbal, three.

Lego-mad Stanley, of Halesowen, West Midlands, is desperate for Kuvan as it would mean he won’t have to consume a synthetic protein drink. The foul-tasting “shake” is packed full of amino acids that PKU sufferers can safely digest. But it smells and tastes almost fishy.

Stanley’s mum Jessica, who is a teacher, told us: “He told me recently, ‘I hate having PKU!’ It’s so tough on him.

“Pre-Covid we could not eat out often and if we did we would have to ring up to ask about ingredients.

“We also have to make sure he doesn’t eat his friends’ food or treats – which makes him feel especially different.

“Why is Britain failing to offer treatment that is available in so many other countries?

“My little boy needs it.

“If the NHS could get Kuvan it would be brilliant.

“It would give Stanley the freedom to enjoy food and take away a huge level of stress from us as a family.

“Also I would love him to be able to eat natural protein, instead of synthetic supplements, which would be far better quality and better for his long-term development.”

Two-year-old Willow is desperate to eat what her non-PKU older sister Grace has for meals – but instead she can only eat 3g of protein a day.

Meat, eggs, wheat and dairy products are all banned, which rules out milk, pasta, bread, rice, breakfast cereals, porridge, crackers and pizza.

Instead Willow’s mum Chelsea Glover, 27, from the Wirral, has to measure and scrutinise every single meal and morsel that her daughter eats, often leaving the mother at her wit’s end.

She told us: “Willow’s already noticing her meals are different to Grace’s, and even though we try and make them look similar, in reality they are not.

“Willow will bring us crisps, yoghurts and drinks out of the kitchen and we have to tell her, ‘No, you’re not allowed them.’

“She doesn’t understand why she can’t have them, and the guilt is starting to set in for me.”

For the last 12 years, life-changing Kuvan – BioMarin’s brand name for the PKU drug sapropterin – has been available in European countries including Latvia and Estonia. It costs around £45,000 per adult per year but as little as £4,000 for a toddler.

Despite holding a number of reviews into whether to buy the drug, the NHS has so far failed to strike a deal.

Last month we told how campaigners backed by MPs met NHS England (NHSE) to urge chiefs to finally strike a deal.

BioMarin’s patents expire this month, meaning that in 2021 rival firms can make their own, cheaper versions of the drug, which the NHS could also buy.

If BioMarin refuses to lower its price, campaigners urge NHSE to negotiate a deal with the alternative drug makers instead. Labour MP for Blaydon, Liz Twist, told the Daily Express: “Families need Kuvan now. This has to be an immediate health priority.”

NHSE said: “We have repeatedly urged BioMarin to offer the drug at a fair price, a cost-effective use of taxpayer money.”

BioMarin refused to comment.

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