Results 171 to 180 of about 6,859 (228)

Cerebrospinal fluid biomarkers of efficacy in patients affected by Spinal Muscular Atrophy type 1 treated with nusinersen. [PDF]

Maria Sframeli, Francesca Polito, Gian Luca Vita, Vincenzo Macaione, Eloisa Gitto, Gian Luca Vita, M. Aguennouz, Sonia Messina   +7 more
openalex   +1 more source

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models [PDF]

, 2018
et al,, Hoye, Mariah L, Miller, Timothy M, Schoch, Kathleen M, Setnicka, Amy, Shabsovich, Mark, Wegener, Amy J   +6 more
core   +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

Hématome épidural rachidien spontané en cas de dorsalgie aiguë [PDF]

, 2017
Anisau, Aliaksandr, Vangeneugden, Johan, Vanhoenacker, Filip   +2 more
core   +1 more source

Neuronal Pentraxin 2 as a Potential Biomarker for Nusinersen Therapy Response in Adults with Spinal Muscular Atrophy: A Pilot Study. [PDF]

Biomedicines
Neuhoff S, Schmitt LI, Liebig KC, Hezel S, Tilahun NI, Kleinschnitz C, Leo M, Hagenacker T.   +7 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

Comparative Clinical Outcomes of Nusinersen and Gene Therapy in Spinal Muscular Atrophy Type 1.

JAMA Netw Open
Ropars J, Cances C, Garcia-Uzquiano R, Gomez-Garcia de la Banda M, Barnerias C, Audic F, Durigneux J, Halbert C, Nkam L, Laugel V, Espil C, Walther-Louvier U, Davion JB, Isapof A, Le Goff L, Desguerre I, Quijano-Roy S, Grimaldi L, French SMA Registry Study Group.   +18 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

Nusinersen Use in Spinal Muscular Atrophy [PDF]

Pediatrics, 2019
openaire   +2 more sources

Analyse der gesundheitsbezogenen Lebensqualität von Patienten mit spinaler Muskelatrophie (SMA) unter der Therapie mit Nusinersen und Ermittlung der Belastung ihrer pflegenden Angehörigen

Velling, Ann-Kathrin
openalex   +1 more source