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Tuberous Sclerosis Drug Pipeline Analysis Report 2024

Tuberous sclerosis, caused by mutations in the genes TSC1 and TSC2, affects around 1 in 6000 to 1 in 10,000 live births. The overall prevalence of this rare genetic disorder is approximately 1 in 20,000. Currently, there is no cure for tuberous sclerosis and the available treatments are symptomatic and supportive including early development interventions for reducing the risk o...

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