Results 141 to 150 of about 19,843 (235)

Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment. [PDF]

Orphanet J Rare Dis, 2021
Monnette A, Chen E, Hong D, Bazzano A, Dixon S, Arnold WD, Shi L.   +6 more
europepmc   +1 more source

Evaluation of putative CSF biomarkers in paediatric spinal muscular atrophy (SMA) patients before and during treatment with nusinersen. [PDF]

J Cell Mol Med, 2021
Johannsen J, Weiss D, Daubmann A, Schmitz L, Denecke J.   +4 more
europepmc   +1 more source

Activation of Muscle-Specific Kinase (MuSK) Reduces Neuromuscular Defects in the Delta7 Mouse Model of Spinal Muscular Atrophy (SMA). [PDF]

Int J Mol Sci, 2021
Feng Z, Lam S, Tenn ES, Ghosh AS, Cantor S, Zhang W, Yen PF, Chen KS, Burden S, Paushkin S, Ayalon G, Ko CP.   +11 more
europepmc   +1 more source

Executive function is inversely correlated with physical function: the cognitive profile of adult Spinal Muscular Atrophy (SMA). [PDF]

Orphanet J Rare Dis, 2021
Mix L, Schreiber-Katz O, Wurster CD, Uzelac Z, Platen S, Gipperich C, Ranxha G, Wieselmann G, Osmanovic A, Ludolph AC, Petri S, Lulé D.   +11 more
europepmc   +1 more source

Pregnancy in women with spinal muscular atrophy (SMA): maternal and neonatal outcomes with multi-speciality management. [PDF]

J Neurol
Cohen D, Nana M, Hart N, Arrandale L, Kelleher C, Nelson-Piercy C.   +5 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

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

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

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