Results 71 to 80 of about 3,596 (160)

Nusinersen for spinal muscular atrophy [PDF]

Therapeutic Advances in Neurological Disorders, 2018
Claudia D. Wurster, Albert C. Ludolph
openaire   +3 more sources

A changing early onset scoliosis phenotype in spinal muscular atrophy: nusinersen treatment is associated with younger age, increased curve magnitude, and kyphosis at the time of index surgery. [PDF]

Spine Deform
Hollyer I, Generoso T, Young B, Bryson X, Segovia N, Albarran M, Vaughan M, Mcintosh A, Ramo B, Bouton D, Snyder B, Vitale M, Smith J, Roye B, Cahill P, Brooks J, El-Hawary R, Group PSS, Vorhies JS.   +18 more
europepmc   +1 more source

Machine learning predicts treatment response to nusinersen in non-sitter spinal muscular atrophy (SMA). [PDF]

J Neuromuscul Dis
Stimpson G, O'Reilly E, Coratti G, Ridout D, Chakraborti T, Mitra R, de Sanctis R, Pane M, Scoto M, Mercuri E, Muntoni F, Baranello G, International SMA Consortium.   +12 more
europepmc   +1 more source

Neurofilaments in cerebrospinal fluid and/or serum as a possible biomarker of pediatric spinal muscular atrophy. [PDF]

J Neuromuscul Dis
Kumhera M, Vrsčaj E, Loboda T, Osredkar D, Holotová P, Rohlenová M, Lauerová B, Jurášová V, Hanzalová J, Pavlíková M, Haberlová J.   +10 more
europepmc   +1 more source

Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations. [PDF]

Front Pharmacol
Belančić A, Maria Gkrinia EM, Lam YW, Vitezić D.   +3 more
europepmc   +1 more source

Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances. [PDF]

Ther Adv Neurol Disord
René CA, Parks RJ.
europepmc   +1 more source

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

Serum circulating cell-free messenger RNA profile response to risdiplam treatment in adult patients with late-onset spinal muscular atrophy. [PDF]

Brain Commun
Leo M, Schmitt LI, Liebig KC, Hezel S, Neuhoff S, Roos A, Ruck T, Kleinschnitz C, Hagenacker T.   +8 more
europepmc   +1 more source

Gene therapy benefits in a small cohort of symptomatic 5q spinal muscular atrophy patients and real world pediatric outcomes. [PDF]

J Pediatr (Rio J)
Santos ARBV, Ferreira LS, Santos TS, Mazzeu JF, Sorte NCAB.   +4 more
europepmc   +1 more source