Chapters authored
Novel Therapeutic Approaches for Skeletal Muscle Dystrophies
Muscular dystrophies (MDs) are inherited diseases that affect skeletal and cardiac muscle tissues. Cases range from mild to very severe, resulting in respiratory or cardiac failures. No cures are available for MDs and corticosteroid treatments, mainly deflazacort and prednisolone, only help to control the inflammatory process and slightly delay the progression of the disease. This is due to the beneficial effect on pulmonary function and scoliosis. Walkers and wheelchairs are used to strengthen patients’ independence and walking ability. When respiratory and/or cardiac muscles become weak, mechanical ventilation is mandatory. In addition, hypertension, cataracts, excessive weight gain and vertebral fracture are often serious side effects of deflazacort and prednisolone treatments.
Part of the book: Muscle Cell and Tissue
Advanced Treatments and Emerging Therapies for Dystrophin- Deficient Cardiomyopathies
Dystrophinopathies are characterized by skeletal and cardiac muscle complications because of a lack or shortened DYSTROPHIN protein. Ventilation assistance and corticosteroid treatment have positively affected life outcome but lead to an increased incidence of cardiomyopathy. Cardiomyopathy is now the leading cause of death in patients with dystrophinopathy. Thus, coherent guidelines for cardiac care have become essential and need to be communicated well. Progression of cardiac complications in patients with dystrophinopathy diverges from standard dilated cardiomyopathy development and monitoring and medical care for dystrophinopathy. This chapter summarizes current guidelines and recommendations for monitoring and clinical treatment of cardiac complications in patients with dystrophinopathy and provides a thorough survey of emerging therapies focusing on cardiac outcomes.
Part of the book: Cardiomyopathies