The article discusses an experimental treatment given to an infant girl with a rare genetic disorder, which focused on inhibiting a mutant protein linked to her condition. While the treatment led to a decrease in the number of seizures the infant experienced, the results highlighted a significant limitation: the procedure did not result in any improvement regarding the neurological impairments that were present. This conclusion raises important questions about the overall effectiveness of targeting mutant proteins in pediatric genetic disorders.
The experimental treatment demonstrated considerable success in reducing the frequency of seizures in a baby girl suffering from a rare genetic disorder by targeting a mutant protein.
Despite the reduction in seizures within the treatment cohort, the study revealed that there was no notable improvement in the neurological impairments associated with the condition.
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