Πέμπτη 25 Ιουλίου 2019


First-in-human gene therapy launches for pediatric patient with rare neurogenerative disease 

JoJo
In May, a ten-year-old girl named JoJo received gene therapy for an extremely rare neurogenerative disease called GM1 gangliosidosis. GM1 progressively destroys nerve cells (neurons) in the brain and spinal cord, leading to muscle and movement weakness, difficulty swallowing and a shortened life expectancy. 

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