Rescue is so close - help create a drug for children with SPG47
Campaign goal: Financing research for the gene therapy development
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Campaign goal: Financing research for the gene therapy development
Campaign description
GENE THERAPY is the only hope for children with SPG47! Your support is needed!
There are children who want to move, play, or just stand for the first time, but their bodies get weaker every day. Those are little patients with spastic paraplegia - a progressive, ruthless genetic disease.
SPG47 is a rare, genetic, neurodevelopmental disorder that causes progressive damage to the nervous system, causing muscle weakness and spasticity, and developmental delay. Children slowly lose the ability to perform even basic activities…
For parents, the diagnosis is the beginning of a life in constant fear for their child’s every step, word, or breath. Little ones learn how to function in a world that becomes difficult for them. In many cases, they end up losing independence and require the use of a wheelchair!
The worst part is that treatment for SPG47 hasn’t been invented yet. The disease is unstoppable, taking the childhood away bit by bit… Unfortunately, because of its rarity, the pharmaceutical companies find it simply unprofitable to develop a drug for it. As a result, it is the parents who must fight for their children’s future, and they will do everything to save them.
The last resort for these families is gene therapy, which might be a dream come true. The Boston Children’s Hospital has been preparing for the gene therapy clinical trials! This breakthrough solution can stop the disease progression and even let children with SPG47 maintain their physical abilities!
The treatment includes administering the missing gene to a child’s nervous cells and stopping the disease. However, several million dollars are still needed to start the clinical trials. Without these funds, the gene therapy cannot be started…
It’s a race against time. Month by month, children lose the previously acquired developmental milestones and independence… This is why we have started a fundraiser to support the research financing.
We are asking you from the bottom of our hearts to help stop this disease and give children a chance to regain health. Every donation brings those little patients closer to a happy childhood and a better tomorrow! Thank you for your support and commitment!
The Siepomaga Foundation
