Dear Peter Lilley MP
I would be grateful of you assistance in gaining approval for the drug Everolimus to be made available under the UK rare disease strategy.
My son; Leo who is currently 12, was born with the genetic condition Tuberous Sclerosis, a condition that effects 1 in every 6000 live births. The condition has caused him to have Epilepsy, Autism and Learning difficulties due to growths in his brain, kidneys and other areas of his body.
In 2014 he was placed on a trial drug; Everolimus, which has substantially reduced his seizures and the severity of the seizures that remain. His skin lesions have reduced greatly so that he doesn’t experience spontaneous bleeding. His quality of life has improved and he is more able to engage with society…it is somewhat of a wonder drug.
Unfortunately the drug is not yet available through the NHS and Leo is reliant on the generosity of the drugs company; Novartis, to provide his medication free. Many TS sufferers have not been as fortunate and do not have access to this drug, which as I’m sure you can appreciate is heartbreaking.
The Tuberous Sclerosis Association (https://www.tuberous-sclerosis.org) is currently working with the NHS England to make this treatment available to children with TS however they need our support to gain a positive outcome. Please could you use you considerable influence to help them make Everolimus available to Tuberous Sclerosis sufferers.
If you need any further information, then do not hesitate to contact me.