I have Hereditary Spastic Paraplegia-52, or SPG52, an extremely rare neurodegenerative disorder.
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A gene therapy cure is almost ready for clinical trail - but it can't move forward without funding.
Me and my friends need your help to make it a reality before it's too late!
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Thank you for giving us hope for a brighter future!
What is SPG52?
Spastic Paraplegia 52 is extremely rare neurodegenerative disorder that affects children. ​The disorder causes:
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global developmental delay
significant intellectual disabilities
poor or absent speech
seizures
progressive spasticity (stiffness) in limbs
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It slowly takes away the child's ability to walk, then potentially the use of their arms and hands.​
However, there is hope.
Gene therapy is within reach...but it's most effective in younger children...and it needs funding.
Help fund the cure and be a light for Leo.
Where the Research Stands
The research team at Universitat Autònoma de Barcelona (UAB) has developed a gene therapy approach for SPG52.
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They have completed the first preclinical trials in cell models and mice. These studies show that the gene therapy can correct faulty neuronal signaling, improve motor coordination, and restore neural-to-muscle communication.
They are now in the process of designing safety and toxicity studies. After these studies, they will request regulatory approval from the Spanish Agency for Medicines and Health Products to begin a clinical trial.
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In short: we are nearing the threshold for a human trial. But we need funds now to cross it.
What is Needed
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