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Jotrol overcomes these barriers using patented micellar technology, enhancing bioavailability and enabling therapeutically effective doses. CAMP4’s RNA-based therapies focus on geneticdiseases like urea cycle disorders, while Rapport’s small molecules aim to address epilepsy, pain and bipolar disorder.
CDKL5 deficiency disorder (CDD) is a rare genetic condition caused by mutations in the CDKL5 gene, which is essential for brain development and function. They are exploring a prodrug formulation of ganaxolone to potentially enhance the patient experience and treatment effectiveness by aiming to improve the drug’s bioavailability.
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