U.S., Jan. 16 -- ClinicalTrials.gov registry received information related to the study (NCT07344480) titled 'Retrospective Natural History Study of RASopathy-associated Cardiomyopathy' on Jan. 07.
Brief Summary: RASopathy-associated hypertrophic cardiomyopathy (RAS-CM) is a disease with high morbidity and high mortality if presenting during infancy. Targeted therapies have shown significant activity in preclinical models and case reports. Drugs that target the underlying cause of this disease are now developed in cancer patients. Conducting randomized trials is not possible in severely ill infants with RAS-CM. Existing historical controls from older eras are not sufficient as external controls to support drug development as they lack criti...