Mother goes straight to the top to access help for son

Mother goes straight to the top to access help for son

A MOTHER from Tunbridge Wells has taken her fight to secure life-changing treatment for her young son to the Houses of Parliament.

Six-year-old George Tappenden suffers from the muscle wasting condition spinal muscular atrophy (SMA) Type 2.

His mum Lucy went to Parliament earlier this month to attend Muscular Dystrophy UK’s inaugural Fast Track event.

This brought together people with muscular dystrophy, their MPs, representatives from the pharmaceutical industry and the National Institute for Health and Care Excellence (NICE).

Lucy and other families heard how lengthy and unnecessary delays to the UK’s drug approval process is putting lives at risk.

Many muscle-wasting conditions progress quickly, and the Fast Track campaign is calling for early-access schemes to bring  treatments to patients sooner.

It is also calling for the NHS to be given more powers to negotiate deals with pharmaceutical companies in order to streamline the availability of treatments.

And Fast Track wants a more efficient NICE approval process for rare disease drugs such as Spinraza, the first and only treatment for SMA.

Under the scheme Spinraza could be made available to George and other families while NICE is assessing it for funding on the NHS.

For babies with the most severe SMA type, life expectancy without access to the drug is rarely more than two years.

Lucy said: “SMA is a cruel condition that takes away your ability to do the things we all take for granted.

“Knowing there is a treatment out there that could make life a little easier for George has given me renewed hope.

“Getting access to Spinraza could be lifechanging for him. My fight won’t stop until I
secure a brighter future for my son.”

Nic Bungay, Director of Campaigns, Care and Information at Muscular Dystrophy UK, said: “For families like Lucy’s, who live with the effects of muscle-wasting conditions, every day counts.

“Time and again we’ve seen how the UK lags behind other countries in Europe when it comes to getting life-saving treatments to patients as quickly as possible.

“With new treatments for muscular dystrophy on the horizon, we must tackle the barriers that prevent them getting to patients like George.”

For more information on the campaign, visit

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