How TMC’s strategic regulatory support advanced a rare disease treatment for CDKL5 CASE STUDY Background & objectives Cyclin-dependent kinase-like 5 deficiency disorder (CDKL5) is a rare, debilitating genetic condition that primarily affects females and presents in the first few months of life. The patient population is extremely limited, with an estimated prevalence of 1 in 30,000 to 1 in 40,000 live births. Given the lack of treatment options, there’s a significant unmet need for novel therapies to address CDKL5 and improve the lives of affected patients. TMC, a global clinical research organisation, was engaged by a bio-pharmaceutical client aiming to develop a novel treatment for CDKL5. Cause Mutations in the CDKL5 gene, which affects the development and function of the brain Symptoms • Severe developmental delay • Motor impairment • Autism spectrum disorder-like characteristics • Epilepsy with multiple seizures daily (these are often resistant to treatment and can lead to further developmental regression) Prognosis • Significantly impaired quality of life • Shortened lifespan due to complications from seizures and associated medical issues Treatment options No cure, and limited treatment options

How TMC’s strategic regulatory support advanced a rare disease treatment for CDKL5

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