As you probably know, the RAINBOWFISH study included pre-symptomatic children with spinal muscular atrophy, mainly identified through newborn screening and some by family history, and the study revealed that after three years, the absolute majority of children reached the milestones of proper neurological development. And actually, the RAINBOWFISH study is the first study which also covers the cognitive functions of the children...
As you probably know, the RAINBOWFISH study included pre-symptomatic children with spinal muscular atrophy, mainly identified through newborn screening and some by family history, and the study revealed that after three years, the absolute majority of children reached the milestones of proper neurological development. And actually, the RAINBOWFISH study is the first study which also covers the cognitive functions of the children. And we could prove that all of the children developed well in terms of cognition and the functions which can be measured with cognitive tests. They can all also swallow and talk and breathe and they do not need any support except one child who is using only breathing help when they have some infection or coughing or something like this. And the motor milestones are reached. So we are very happy to have those results and were able to present them. And actually, only two children did not develop as expected, but they were the children from which we presented at the beginning the weakest C-map, which was lower than 1.5 microvolts, and that probably raises the question of whether those children were really pre-symptomatic or maybe they developed already during pregnancy some motor impairment, in terms of which was not seen after birth, but probably some damage in the muscles or in the nerves was already there.
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