Patients with muscular dystrophies and their caregivers, including families and clinical teams, face significant challenges. These individuals often experience progressive difficulties in breathing and sleeping, along with decreased exercise tolerance due to cardiorespiratory insufficiency. This takes a substantial toll on them and their families. The purpose of this NOSI renewal is to enhance basic, translational, and clinical research in the cardiorespiratory and sleep complications associated with muscular dystrophies. The goals include elucidating phenotypes, optimizing metrics for early diagnosis, refining therapies such as gene therapy delivery to cardiac and respiratory muscles, and exploring mechanisms and treatment strategies for sleep-disordered breathing (SDB).

Muscular dystrophies are a heterogeneous group of congenital diseases, including Duchenne muscular dystrophy (DMD), facioscapulohumeral muscular dystrophy (FSHD), and myotonic dystrophy (DM), all leading to progressive

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