ATP13A3 and pulmonary arterial hypertension: Although the safety of AMXT 1501 (in combination with DFMO) has been established in phase I/II clinical trials (NCT03536728; NCT05500508), targeting ATP13A3 with specific inhibitors also may not be without risk, since ATP13A3 gene mutations have been associated with the pathogenicity of pulmonary arterial hypertension [32, 45].