Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study. [PDF]
Gavriilaki M +4 more
europepmc +1 more source
Diagnostic Journey of Korean Patients with Spinal Muscular Atrophy. [PDF]
Kim SH +6 more
europepmc +1 more source
Spinal adhesive arachnoiditis in an adult patient with spinal muscular atrophy type 3 treated with intrathecal therapy. [PDF]
Ubysz J +3 more
europepmc +1 more source
A self-reported Brazilian registry of 5q-spinal muscular atrophy: data on natural history, genetic characteristics, and multidisciplinary care. [PDF]
Mendonça RH, Godoi JSA, Zanoteli E.
europepmc +1 more source
A Clinical and Pathological Study of an Unusual Case of Progressive Muscular Dystrophy - Type Facio-Scapulo-Humeral - with Special Reference to its Nosology and Pathogeny [PDF]
Marshall, R. M
core
Clinical and Genetic Profiles of 5q- and Non-5q-Spinal Muscular Atrophy Diseases in Pediatric Patients. [PDF]
Nishio H +7 more
europepmc +1 more source
Reduced white matter integrity and disrupted brain network in children with type 2 and 3 spinal muscular atrophy. [PDF]
Nie H +11 more
europepmc +1 more source
The long-term efficacy of nusinersen on respiratory functions in children with symptomatic spinal muscular atrophy type 1. [PDF]
Kiliç MA +9 more
europepmc +1 more source
Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China. [PDF]
Peng J +20 more
europepmc +1 more source

