GOAL: Gene therapy with Zolgensma - the only chance to save the life of Patryk Radwański, 7 months

Lives: Wrocław, Poland

Diagnosis: SMA (spinal muscular atrophy) type 1, the most severe form.

My friend's son Patryk was born in April with a genetic disease known as SMA or Spinal Muscular Atrophy type 1. The disease gradually takes away the afflicted’s strength and motor skills down to the ability to move. Patryk’s only chance is an expensive gene therapy conducted in the US. It stops the disease from developing, gradually gives back some of the lost motor functions. The only thing standing in Patryk's parent's way is its unimaginably high cost. Children afflicted on SMA type 1 usually do not make it to age two. Patryk is able to lift his arms and twist his head, but these are the only movements he’s capable of. The disease didn’t take those – yet. Up until very recently, SMA had been incurable. As it stands, contemporary medicine does give him a chance! Thankfully, the first available SMA drug - Spinraza - is refunded in Poland. Spinraza slows down the disease’s advancement. Unfortunately, the drug affects each child individually, which makes its ultimate effects difficult to predict. It can also have no effect. Unfortunately, this happened to little Patryk. After the 2nd dose, this small warrior stopped eating independently and has been fed through a nasogastric tube ever since.

But there’s still hope! There’s a new treatment in the US which was proven to stop the disease’s advancement. It also gives back lost motor functions! The drug supplies a copy of the particular gene that Patryk is missing – the one causing his muscles to atrophy. As a result, Patryk’s organism would begin producing proteins, enabling his muscles to function. This means there’s a chance he could move, sit, eat – he could live normally. That's why your help is so greatly needed to save this little boy`s life.

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