Results 161 to 170 of about 7,657 (243)

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

Clinical Experience of Timing Treatment in Newborns with Spinal Muscular Atrophy: A Call for Standardized Screening Practices in Italy. [PDF]

Int J Neonatal Screen
Bitetti I, Iannaccone R, Margiotta G, Varone A.   +3 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

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

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

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

Application of Biomarkers in Spinal Muscular Atrophy. [PDF]

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

Case Report: Spinal muscular atrophy with IgA nephropathy: a coincidence or association? [PDF]

Front Pediatr
Gu Y, Wang L, Yuan X, Han Y, Han P, Cui J, Wang X, Huang Y, Zhang L, Li C.   +9 more
europepmc   +1 more source

The 2025 Oligo Meeting in Budapest: Highlights of the 21^st annual meeting of the oligonucleotide therapeutics society. [PDF]

Mol Ther Nucleic Acids
Godinho BMDC, Guo S.
europepmc   +1 more source