The goal of the proposed project is to determine the safety and tolerability as well as the
preliminary efficacy of a novel small molecule drug, S48168 (ARM210), for the treatment of
Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT1). This disease is associated
with fatal changes in heart rhythms leading to sudden death with exercise or excessive
excitement. It is due to mutations in the Ryanodine Receptor calcium release channel, which
cause leaky channels leading to the disease. S48168 (ARM210) repairs these leaky channels and
can be a disease-modifying therapy restoring normal function to the channels. This result
would allow children and adults with CPVT to live normal, active lives.