Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in
2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved …

Abstract Duchenne Muscular Dystrophy (DMD) is a severe, life-limiting and incurable
condition. However, studies estimating quality of life and those measuring actual quality of …

Steadily improving management of Duchenne muscular dystrophy (DMD) continues to lead
to improved physical and functional status, allowing increasingly successful transitions to …

Abstract Introduction/Aims Risdiplam is the newest available treatment for patients with
spinal muscular atrophy (SMA). There is little information on its use in adults. We present the …

Background Living with a chronic disease often means experiencing chronic treatments and
regular multidisciplinary monitoring as well as a profound life-changing experience which …

The positive outcome of different therapeutic approaches for spinal muscular atrophy (SMA)
in clinical trials and in clinical practice have highlighted the need to establish if functional …

Health Sciences, Centre de recherche Charles-Le-Moyne, Université de Sherbrooke, Centre
intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean, 2230 de …

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common genetically
inherited myopathies in adults. It is characterized by incomplete penetrance and variable …

Objective This systematic review aims to provide an overview of instruments used to assess
behavioral and psychosocial functioning of patients with Duchenne and Becker muscular …

Hereditary motor and sensory neuropathy (HMSN) and myotonic dystrophy (MD) are chronic
neuromuscular diseases that cause progressive muscular impairment and impact patient's …