Results 81 to 90 of about 3,596 (160)

Therapeutic strategies for spinal muscular atrophy: the history and future perspective. [PDF]

Front Hum Neurosci
Liu J, Liu Y, Yuan A, Ning K.
europepmc   +1 more source

Modeling the Budget Impact of Disease-Modifying Therapies in Infantile-Onset Spinal Muscular Atrophy. [PDF]

Clin Drug Investig
Abuloha S, Goodin A, Jiao T, Corti M, Svensson M.   +4 more
europepmc   +1 more source

Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions. [PDF]

Front Mol Med
Sexton MM, Crow EW, Peng CA.
europepmc   +1 more source

Lung Function and Respiratory Muscle Strength in Symptomatic Children and Adults With Spinal Muscular Atrophy Treated With Nusinersen. [PDF]

Neurology
Vercoelen F, Arts LAP, de Bruijne MEM, van Eijk RPA, Vermeer LM, Wadman RI, Verweij-van den Oudenrijn LP, Zoodsma RS, Cuppen I, Bartels B, Asselman FL, Wijngaarde CA, van der Pol WL, Veldhoen ES.   +13 more
europepmc   +1 more source

Effect of nusinersen on respiratory function in patients with spinal muscular atrophy: an 18-month single-center prospective study. [PDF]

Front Pediatr
Luo T, Xu L, Tang W, Huang L, Li Y, Yue Z.   +5 more
europepmc   +1 more source

From Genes to Function: Clinical Experience with the Effectiveness and Safety of Risdiplam and Nusinersen in Spinal Muscular Atrophy. [PDF]

Iran J Child Neurol
Sedighi P, Fayyazi A, Hosseini F, Karimi K, Esfahani H, Mahdi Hosseiny SM.   +5 more
europepmc   +1 more source

Extracellular vesicles as biomarkers of disease progression and therapeutic response in patients with spinal muscular atrophy. [PDF]

Mol Ther Adv
Poulin KL, René CA, Smith IC, Vacratsis PO, Burger D, McMillan HJ, Kothary R, Perkins TJ, Warman-Chardon J, Parks RJ.   +9 more
europepmc   +1 more source

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

Immune Cell-Specific SMN Expression and Sustained Immune Alterations in Treated Spinal Muscular Atrophy. [PDF]

J Clin Invest
Tapken I, Rahmel-Stein K, Ehlers C, Cieri F, Detering NT, Schüning T, Bjelica B, Mindermann C, Neuhoff S, Schmitt LI, Leo M, Hagenacker T, Illsinger S, Di Schiavi E, Graalmann T, Petri S, Kalinke U, Claus P.   +17 more
europepmc   +1 more source

Clinical Characterization of Patients With 5q Spinal Muscular Atrophy Types 2 and 3 in Brazil: A Cross-Sectional Observational Study. [PDF]

Clin Genet
Batista EC, Zanoteli E, Fonseca HAR, Polido GJ, Saute JAM, Ortega AB, Junior MCF, Oliveira ASB, de Souza PVS, Giannetti JG, Pessoa ALS, de Queiroz Campos Araújo AP, Neves MFT, da Silva RTB, Monfardini F, Dos Santos GP, Dos Santos Sampaio B, Moia DF, Júnior FG, de Piano LPA, de Albuquerque CSN, Duarte FM, Aparecida Rodrigues Sant'Anna V, Pereira M, de Bortoli Livramento J, Vicente Pereira Soares R, Pedrosa DA, Teich V, Rizzo LV, Berwanger O, de Andrade JBC, Silva GS.   +31 more
europepmc   +1 more source