Heart

Heart Heart Failure with Preserved Ejection Fraction
Patients with Heart Failure with Preserved Ejection Fraction (HFpEF) have grown in number and currently comprise ~50% of the overall heart failure population. HFpEF patients have debilitating symptoms such as fatigue, weakness, shortness of breath with minimal exertion or at rest, and they experience high incidence of morbidity and mortality. HFpEF is an age-related condition with patients experiencing serious comorbid conditions once they present with HF. There are very few effective treatments for HFpEF and no disease modifying therapies. There is an urgent need for novel targeted therapies that reverse the pathophysiology of HFpEF.

Heart Failure with Reduced Ejection Fraction

Heart failure with reduced Ejection Fraction (HFrEF) is a complex pathophysiological syndrome encompassing a range of abnormalities that impair normal cardiac function and result in inability of the heart to pump a sufficient supply of blood to meet the metabolic requirements of the body, or to do so only at elevated ventricular filling pressures. Once established, HFrEF is generally progressive, irreversible, associated with debilitating symptoms, frequent re‑hospitalizations and high mortality rates.

HFrEF treatment is tailored to the individual patient, based on the underlying causes, comorbidities and the patient’s stage at diagnosis and NYHA functional classification. Treatment goals are to reduce symptoms, prolong survival, improve the quality of life and slow or prevent disease progression. Despite optimal medical therapy employing a wide range of pharmacologic, device and surgical therapeutic options, over the long‑term most patients experience a progressive decline, ultimately succumbing to the syndrome or one of the underlying contributing conditions. There is an urgent need for disease modifying therapies for patients with HFrEF.

Duchenne Muscular Dystrophy-associated Cardiomyopathy

Duchenne muscular dystrophy (DMD) is a rare, life-threatening, genetic, sex-linked disorder affecting all skeletal as well as cardiac muscle. It is the most common and severe form of muscular dystrophy among children and is caused by a deficiency of dystrophin, DMD presents initially with muscle weakness, progressive weakening of the pulmonary muscles, kyphosis and scoliosis, and in the second and third decade of life progressive cardiomyopathy and resultant symptomatic heart failure. DMD induced cardiomyopathy is the main cause of mortalilty in DMD patients. There are few effective therapies for DMD cardiomyopathy and these rely on standard, generic approaches to heart failure with reduced ejection fraction. Therefore, development of new approaches better targeting the pathophysiology of DMD could result in an improvement in quality of life and potentially prolonged survival providing what is currently a substantial unmet medical need.