Results 171 to 180 of about 888,799 (202)

Risdiplam therapy in adult patients with Spinal muscular Atrophy (SMA). A 24-month-real-world experience at a single muscular centre. [PDF]

Acta Myol
Cesarone E, Picillo E, Scutifero M, Passamano L, Nigro V.   +4 more
europepmc   +1 more source

The incidence of hydrocephalus among patients with and without spinal muscular atrophy (SMA): Results from a US electronic health records study. [PDF]

Orphanet J Rare Dis, 2021
Viscidi E, Wang N, Juneja M, Bhan I, Prada C, James D, Lallier S, Makepeace C, Laird K, Eaton S, Dilley A, Hall S.   +11 more
europepmc   +1 more source

Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study

, 2017
Nataliia Sych, 1 Mariya Klunnyk,1 Iryna Matiyashchuk,1 Mariya Demchuk,1 Olena Ivankova,1 Andriy Sinelnyk,1 Khrystyna Sorochynska,2 Marina Skalozub3 1Clinical Department, 2Stem Cells Bank, 3Laboratory and Biotechnology Department, Cell Therapy Center ...
Sych N, Matiyashchuk I, Sorochynska K, Klunnyk M, Sinelnyk A, Skalozub M, Ivankova O, Demchuk M   +7 more
core  

Awareness screening and referral patterns among pediatricians in the United States related to early clinical features of spinal muscular atrophy (SMA). [PDF]

BMC Pediatr, 2021
Curry M, Cruz R, Belter L, Schroth M, Lenz M, Jarecki J.   +5 more
europepmc   +1 more source

Respiratory function in 192 adult patients with spinal muscular atrophy (SMA) treated with nusinersen - a multicenter observational study. [PDF]

Orphanet J Rare Dis
Wurster CD, Stolte B, Kessler T, Freigang M, Bjelica B, Ilse B, Koch JC, Cordts I, Mensch A, Zeller D, Uzelac Z, Sam G, Lapp HS, Wohnrade C, Rödiger A, Muhandes MT, Schneider I, Bellut J, Nentwich J, Dorst J, Schuster J, Schreiber-Katz O, Osmanovic A, Totzeck A, Thimm A, Steinbach R, Grosskreutz J, Kleinschnitz C, Ludolph AC, Deschauer M, Kirschner J, Dreyhaupt J, Wollinsky K, Petri S, Weiler M, Hagenacker T, Günther R.   +36 more
europepmc   +1 more source

Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scale. [PDF]

J Neuromuscul Dis
Lefton-Greif MA, Belter L, Jarecki J, Schroth M, Zeng Y, Crawford TO, Eaton C.   +6 more
europepmc   +1 more source

A perioperative nursing care protocol for patients with spinal muscular atrophy (SMA) type II or type III undergoing spinal surgery: a 4-year experience in 24 patients. [PDF]

Orphanet J Rare Dis
Li G, Xu K, Liu D, Wu N, Zhang TJ, Chen Y.   +5 more
europepmc   +1 more source

Multi-omics profiling in spinal muscular atrophy (SMA): investigating lipid and metabolic alterations through longitudinal CSF analysis of Nusinersen-treated patients. [PDF]

J Neurol
Zandl-Lang M, Züllig T, Holzer M, Eichmann TO, Darnhofer B, Schwerin-Nagel A, Zobel J, Haidl H, Biebl A, Köfeler H, Plecko B.   +10 more
europepmc   +1 more source

Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen. [PDF]

Int J Mol Sci
Andrés-Benito P, Vázquez-Costa JF, Ñungo Garzón NC, Colomina MJ, Marco C, González L, Terrafeta C, Domínguez R, Ferrer I, Povedano M.   +9 more
europepmc   +1 more source

Design of a Non-Interventional Study to Validate a Set of Patient- and Caregiver-Oriented Measurements to Assess Health Outcomes in Spinal Muscular Atrophy (SMA-TOOL Study). [PDF]

Neurol Ther, 2021
Madruga-Garrido M, Vázquez-Costa JF, Medina-Cantillo J, Brañas M, Cattinari MG, de Lemus M, Díaz-Abós P, Sánchez-Menéndez V, Terrancle Á, Rebollo P, Maurino J.   +10 more
europepmc   +1 more source