Results 111 to 120 of about 4,580 (188)

Nusinersen for type-III spinal muscular atrophy: a 12-month retrospective study in a Brazilian cohort. [PDF]

Arq Neuropsiquiatr
Van Der Linden V, Calado APM, Van Der Linden G, Valença JVDP, Garrido MA, Oliveira KLS, Kerstenetzky MS, Oliveira T.   +7 more
europepmc   +1 more source

Development and Validation of an Ion-Pair Reverse-Phase High-Performance Liquid Chromatography-Electrospray Ionization Mass Spectrometry Method for Determination of Purity of Nusinersen for Quality Control of Drug Substance or Drug Product. [PDF]

Int J Mol Sci
Samoilov M, Zubareva E, Degterev M.
europepmc   +1 more source

Cognitive and neurodevelopmental outcomes in spinal muscular atrophy: a scoping review. [PDF]

Front Cell Neurosci
Gnazzo M, Pisanò G, Baldini V, Giordani S, Caiazza L, Piccolo B, Turco E, Esposito S, Pera MC.   +8 more
europepmc   +1 more source

Delayed diagnosis and genetic testing in spinal muscular atrophy: a case series. [PDF]

Ann Med
Guo X, Luo Y, Li M, Li C, Chang Z, Zhu S, Wang Q, Huang Y.   +7 more
europepmc   +1 more source

Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy. [PDF]

Muscle Nerve
Koutsioumpa C, Darras BT, Griggs RC, Roy B.   +3 more
europepmc   +1 more source

Administration practice and adherence of nusinersen in adults with 5q-spinal muscular atrophy in China: an ambispective multicenter study. [PDF]

BMC Neurol
Yao X, Dai Y, Zhu W, Zhao Y, Shang H, Meng Q, Liu Y, Yu L, Hong D, Chen J, Shen L, Chen W, Chen K, Zhang L, Mai M, Cui L.   +15 more
europepmc   +1 more source

Longitudinal motor function and biomarker correlates in treated adult spinal muscular atrophy: a single-center cohort study. [PDF]

Front Neurol
Yano C, Ando M, Yoshimura A, Higuchi Y, Noda Y, Yuan JH, Nakamura T, Hobara T, Nagatomo R, Kojima F, Yuji M, Hiramatsu Y, Nozuma S, Hashiguchi A, Sakiyama Y, Takashima H.   +15 more
europepmc   +1 more source

Nusinersen effectiveness and safety in children with type II/III 5q-spinal muscular atrophy: Second interim analysis from a Chinese disease registry. [PDF]

Chin Med J (Engl)
Wang X, Yao X, Peng J, Luo R, Lu H, Liang J, Jin R, Wu L, Zhong J, Yang L, Wu J, Hong S, Ruan Y, Shen T, Zhang X, Mao S, Hu J, Wang H, Sun D, Tao Z, Yang G, Bian G, Lyu J, Zhang L, Zhu J, Wang Y.   +25 more
europepmc   +1 more source

Clinical evaluation of ThecaFlex DRx, a novel implantable catheter-port for intrathecal nusinersen delivery in spinal muscular atrophy: Initial results from the PIERRE-IDE study. [PDF]

J Neuromuscul Dis
Mesina-Estarrón I, Abruzzo M, Gomes FC, Thaxton M, Yengo-Kahn A, Stone S, Northam W, Snyder B, Cahill P, Bauer D, Muhonen M.   +10 more
europepmc   +1 more source

Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients. [PDF]

Front Pediatr
He G, Feng Z, Li N, Wei S, Liu Y, Tang X.   +5 more
europepmc   +1 more source