THE father of a two-year-old Hillingdon girl with a devastating muscle-wasting condition has spoken of his family’s fight for a vital drug to be made available on the NHS.

In February 2017, Sophie Guerra was diagnosed with spinal muscular atrophy Type 2 at the age of one.

As the symptoms progressed, Sophie – now aged two – has lost the fine motor skills she was developing. She never learned to walk or crawl, but could shuffle and roll on the floor.

Sophie was also able to stretch her body, reach out and grab objects and stand on her feet. Now, she has lost the ability to do all of these things.

She has limited head control and her upper body strength has diminished so that she cannot even manage to push herself on a wheelchair. Sophie’s condition has taken a heavy toll on the family, particularly her mother, Tammy, who has become Sophie’s full-time carer.

NICE, the agency that approves the availability of drugs and treatments in this country, is currently assessing Spinraza for delivery on the NHS.

A proposed interim scheme involving pharmaceutical company Biogen is under consideration while the assessment is under way.

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Muscular Dystrophy UK has joined forces with SMA Support UK, The SMA Trust and TreatSMA2 to call for the interim scheme to be implemented as quickly as possible. 

It is also calling for the fundamental problems which hold up the approval of drugs for rare diseases like SMA, to be reformed.

Sophie’s dad, Raul Guerra, 34, said: “When Sophie was diagnosed, our world began to fall apart. All the hopes and dreams we had for our little girl began to fade away. When we heard that NICE was starting a review for Spinraza, our spirits were immediately lifted.

“We know it’s not a cure, but it could give Sophie the chance to do the simple things that other children take for granted, like dressing up and playing on her bed. This would be the biggest blessing for my daughter.”

Around 1,300 children and adults are living with the devastating effects of SMA in this country.

Some children who have received Spinraza have seen their muscle strength improve and have already lived long enough to crawl, and even walk.

There is also evidence of the treatment being effective for children with SMA Type 2 and 3.