Fears over new treatments for rare conditions

GROWING CONCERNS: Layanne and Russell Wright with sons Edward , Sam and Joseph

GROWING CONCERNS: Layanne and Russell Wright with sons Edward , Sam and Joseph

First published in News
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A NORTH-EAST mother fears her three sons may miss out on pioneering new treatments for their rare muscle-wasting condition.

Layanne Wright , 34, of Boldon Colliery, South Tyneside, whose boys - Sam, nine, Edward, six, and Joseph , two - all have Becker muscular dystrophy, gave evidence for a report that MPs will hear today (Tuesday, September 3).

She is worried that changes to NHS drugs funding could deny expensive therapies to people with rare conditions. 

A new genetic therapy, Exon Skipping, may treat life-shortening Duchenne muscular dystrophy - and could kick-start research into the Becker form - but it could cost hundreds of thousands of pounds to administer to young patients. The All Party Parliamentary Group (APPG) wants the Government to re- establish a ring-fenced fund for high-cost treatments.

Ms Wright said: "The condition has really taken hold of Edward, he can't walk very far.

“He gets really tired and we are probably going to have to look into getting a wheelchair this summer.

“We’ve put our all into raising funds to back research into effective treatments – it has helped us to cope.

“With three young boys, the earlier we are able to treat them, the more likely we are able to stop the damage to their muscles.

“As more breakthrough treatments come forward, it would be devastating to see funding issues and red-tape around the assessment of drugs to prevent them from reaching our children, who desperately need them."

Local MP Dave Anderson MP, who chairs the APPG, said: “We urge regulators and commissioners to overcome these obstacles and find a feasible, sustainable way of delivering pioneering rare disease treatments.

“Otherwise they may remain entirely out of patients’ reach.” 

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