Results 171 to 180 of about 6,546 (245)

Advancing personalized spinal muscular atrophy care: matching the right biomarker to the right patient at the right time. [PDF]

J Neurol
Corti S, Ottoboni L, Sansone V.
europepmc   +1 more source

Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements. [PDF]

Cost Eff Resour Alloc
Al-Jedai A, Al-Mudaiheem H, AlSakran A, Bashiri FA, Ghamdi F, Almuhaizea MA, AlSamman A, Awad N, Ojeil R.   +8 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

Disease characteristics and treatment status of genetically confirmed spinal muscular atrophy patients: a cross-sectional survey in China. [PDF]

BMC Neurol
Zhang D, Yao F, Ling C, Jiang S, Yang X, Dai Y.   +5 more
europepmc   +1 more source

Sustainable efficacy of nusinersen in children with later-onset spinal muscular atrophy: a 3-year observational study. [PDF]

World J Pediatr
Feng YJ, Yu YC, Zhao JL, Xu L, Zhao CY, Hua Y, Sheng GX, Chen TT, Zhang Y, Wei LL, Gao F, Mao SS.   +11 more
europepmc   +1 more source

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database. [PDF]

Children (Basel)
Toaz E, Pinto N, Kilner K, Cheon E.
europepmc   +1 more source

Neurofilaments as Biomarkers of the Efficacy of Risdiplam Treatment in Early SMA Phenotypes Diagnosed by Newborn Screening. [PDF]

Children (Basel)
Pitarch-Castellano I, Ñungo-Garzón NC, Aragon-Gawińska K, Ibáñez-Albert E, Vázquez-Costa JF, Sevilla T.   +5 more
europepmc   +1 more source

Nusinersen and spinal muscular atrophies: Where are we in 2020?

Annals of Indian Academy of Neurology, 2020
Rakesh K. Singh, Satish V Khadilkar
openaire   +4 more sources

Application of Biomarkers in Spinal Muscular Atrophy. [PDF]

Int J Mol Sci
Gao C, Zhan Y, Chen H, Deng C.
europepmc   +1 more source

Cerebrospinal fluid metabolomics reveals predictive biomarkers of nusinersen therapy efficacy in type II and type III spinal muscular atrophy patients. [PDF]

Neurol Sci
Li D, Sun N, Wu H, Wang X, Shi Y, Yang L, Huang S, Zhang K, Yang C.   +8 more
europepmc   +1 more source