Results 101 to 110 of about 4,580 (188)

Case Report: Ultra-early nusinersen initiation with pre-procedural spinal ultrasound-assisted intrathecal access in a symptomatic neonate with spinal muscular atrophy. [PDF]

Front Pediatr
Xie N, Sui Y, Zhang Y, Zhang L, Li J, Sun Y, Liu X.   +6 more
europepmc   +1 more source

Long-term persistence, safety and effectiveness of nusinersen in spinal muscular atrophy: a population-based study. [PDF]

J Neurol
Aragon-Gawinska K, Nungo-Garzon NC, Muelas N, Sivera R, Sevilla T, Hervas D, Pitarch-Castellano I, Vazquez-Costa JF.   +7 more
europepmc   +1 more source

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

Proteomics profiling and machine learning in nusinersen-treated patients with spinal muscular atrophy

Aim: The availability of disease-modifying therapies and newborn screening programs for spinal muscular atrophy (SMA) has generated an urgent need for reliable prognostic biomarkers to classify patients according to disease severity.
Bartolucci M., Messina S., Mercuri E., Petretto A., Panicucci C., Principi E., Pane M., Albamonte E., Pedemonte M., D'Amico A., Sansone V. A., Sezerman U., Bertini E., Bruno C., Sahin E., Brolatti N., Casalini S., Baratto S., Pintus S., Sframeli M.   +19 more
core   +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

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

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

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

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