Present at birth, Cardiac Heart Defects (CHD) are structural malformations of the heart that trigger a wide range of crippling cardiac dysfunctions.
Nearly 1 in 100 infants are born with CHD that range from light to severe. In 5% of all cases, the severity of CHD is life-threatening. When available, early corrective heart surgery to repair the defects is the preferred intervention, but it requires an open-heart operation on a very small heart. In some cases, this surgical operation is not an option and a palliative and temporary procedure is elected instead.
One such common palliative procedure is known as Pulmonary Artery Banding (PAB) and is currently used in the USA and Europe in about 1000 cases per year. PAB involves suturing a fixed length of an implantable ‘tape’ around the pulmonary artery to narrow the latter. This device allows to tamper down the abnormal blood pressure and thereby protects the heart and lungs’ functions of the babies. This buys newborns and doctors alike precious time before the babies can undergo the final corrective open-heart surgical procedure.
As a private non-for-profit organisation, we strive to uncover the potential of existing molecules to address severe therapeutic unmet needs in rare diseases, thus giving better chances to existing drugs to reach these underserved patients.