Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy [0.03%]
Eteplirsen治疗可缓解杜氏肌营养不良症患者的呼吸功能下降症状
Navid Khan,Helen Eliopoulos,Lixin Han et al.
Navid Khan et al.
Background: Duchenne muscular dystrophy (DMD) patients experience skeletal muscle degeneration, including respiratory muscles. Respiratory decline in glucocorticoid-treated DMD patients, measured by percent predicted forc...
A S Carr,S Shah,D Choi et al.
A S Carr et al.
Here we describe a patient with genetically confirmed ATTR, a family history of the disease and histological confirmation following carpal tunnel release surgery but no other manifestations. The first major neurological or systemic manifest...
Mahima Kapoor,Alexander M Rossor,Matilde Laura et al.
Mahima Kapoor et al.
Systemic amyloidosis can be hereditary or acquired with autosomal dominant mutations in the transthyretin gene (TTR) being the most common cause of hereditary amyloidosis. ATTRm amyloidosis is a multi-system disorder with cardiovascular, pe...