OCRL and oculocerebrorenal syndrome: Our findings reveal that the phosphoinositide 3-kinase inhibitor alpelisib, which is currently approved for cancer therapy, alleviates the aberrant phosphoinositide balance and actin phenotype associated with the OCRL loss, causing a substantial improvement of the endocytic machinery and absorptive capacity in cellular systems and a humanized mouse model for Lowe syndrome/Dent disease 2.