Results 171 to 180 of about 2,725,444 (278)

Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy. [PDF]

Brain Behav
Bahadır Şenol H, Yıldız G, Polat Aİ, Aydın A, Hız AS, Soylu A, Yiş U.   +6 more
europepmc   +1 more source

Spinal muscular atrophy in Ghanaian children confirmed by molecular genetic testing: a case series. [PDF]

Pan Afr Med J, 2023
Hammond CK, Oppong E, Ameyaw E, Dogbe JA.   +3 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]

BMC Pediatr
Peng J, Yao X, Luo R, Wang X, Wu L, Zhong J, Jin R, Lu X, Liang J, Hong S, Yang L, Zhang X, Mao S, Tao Z, Hu J, Sun D, Wang H, Zhang L, Xia Y, Chen K, Wang Y.   +20 more
europepmc   +1 more source

Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study. [PDF]

Muscle Nerve
Gavriilaki M, Moschou M, Pagiantza M, Arnaoutoglou M, Kimiskidis V.   +4 more
europepmc   +1 more source

Reduced white matter integrity and disrupted brain network in children with type 2 and 3 spinal muscular atrophy. [PDF]

J Neurodev Disord
Nie H, Lan S, Wang H, Xiang P, Yan M, Fan Y, Shen W, Li Y, Tang W, Yang Z, Liang Y, Chen Y.   +11 more
europepmc   +1 more source

Sequential treatment with nusinersen, Zolgensma^® and risdiplam in a paediatric patient with spinal muscular atrophytype 1: a case report. [PDF]

Acta Myol, 2023
Bitetti I, Manna MR, Stella R, Varone A.
europepmc   +1 more source

A self-reported Brazilian registry of 5q-spinal muscular atrophy: data on natural history, genetic characteristics, and multidisciplinary care. [PDF]

Arq Neuropsiquiatr
Mendonça RH, Godoi JSA, Zanoteli E.
europepmc   +1 more source

Clinical and Genetic Profiles of 5q- and Non-5q-Spinal Muscular Atrophy Diseases in Pediatric Patients. [PDF]

Genes (Basel)
Nishio H, Niba ETE, Saito T, Okamoto K, Lee T, Takeshima Y, Awano H, Lai PS.   +7 more
europepmc   +1 more source

Successful pregnancy of an SMA type 3 sitter on Nusinersen therapy - a case report. [PDF]

BMC Neurol
Hiebeler M, Thiele S, Walter MC.
europepmc   +1 more source

Diagnostic Journey of Korean Patients with Spinal Muscular Atrophy. [PDF]

Yonsei Med J
Kim SH, Lee CS, Lee SR, Choi YC, Kim SW, Shin HY, Park HJ.   +6 more
europepmc   +1 more source