Harry has a degenerative genetic motor neurone disease called Spinal Muscular Atrophy Type 3. Harry uses a power wheelchair as he can only walk very short distances and he is too weak to self propel in a manual wheelchair. Harry's entire body is becoming weaker and weaker over time - his legs, arms, hands, neck, ability to swallow can even be affected. We have been told he may completely lose the ability to walk by age 14. A drug called Spinraza/nusinersen has become available in America to treat Spinal Muscular Atrophy. With treatment Harry could carry on walking and his abilities could even improve. Without treatment Harry will continue to get weaker and weaker. This treatment is not available or licensed in the U.K. or anywhere else & the NHS will NOT fund any of his treatment. This is a very invasive treatment where Harry would need to be anaesthetised to have the drug administered into his spine by injection every 4 months. We are trying to raise funds to get Harry over to the USA to see a neurologist and funds to go towards the cost of the drug & administration & hospital fees. Each injection list price is $125,000 - that's $375,000 every year & $750,000 in the first year - as 3 additional loading doses are required to start the therapy. Harry is a beautiful, kind, funny & exceptionally clever little boy. Please help us to help him access the treatment he needs to stop any further deterioration and even improve function.
Thank you let's fight by Harry's side 💙