PLUCKY young Sam Brown has been back at home with his family this week after undergoing a new course of life-enhancing drug treatment.
Six-year-old Sam, a pupil at The Whartons Primary School in Otley, has Morquio Syndrome – an extremely rare, degenerative condition that severely affects growth and mobility.
He has been benefitting from Vimizim for the past three years, under a clinical trial, and parents Simon and Katy described the resumption of his treatment as "a huge relief". Drugs company BioMarin agreed to supply it until a final funding decision is made regarding its long-term funding.
The family is now waiting anxiously to see if the National Institute for Health and Care Excellence will recommend the permanent financing of the drug. The decision is expected on October 28.
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