Results 181 to 190 of about 1,154 (251)

Poster Sessions

HemaSphere, Volume 10, Issue S1, June 2026.
wiley   +1 more source

Ubiquitination Insight from Spinal Muscular Atrophy-From Pathogenesis to Therapy: A Muscle Perspective. [PDF]

Int J Mol Sci
Bolado-Carrancio A, Tapia O, Rodríguez-Rey JC.   +2 more
europepmc   +1 more source

Publication Only

HemaSphere, Volume 10, Issue S1, June 2026.
wiley   +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

Longitudinal data collection in pediatric and adult patients with 5q spinal muscular atrophy in Latin America: LATAM RegistrAME study - a clinical registry study protocol. [PDF]

Einstein (Sao Paulo)
Batista EC, Zanoteli E, Monfardini F, Santos GPD, Silva GS, Berwanger O, Rizzo LV, Fonseca HARD.   +7 more
europepmc   +1 more source

ISEV2026 Abstract Book

Journal of Extracellular Vesicles, Volume 15, Issue S1, June 2026.
wiley   +1 more source

Bulbar function in children with spinal muscular atrophy type 1 treated with nusinersen. [PDF]

Dev Med Child Neurol
Stimpson G, Fanelli L, Conway E, Johnson E, Berti B, Scoto M, Muntoni F, Mercuri E, Baranello G, p‐FOIS/CEDAS and OrSAT Working Group.   +9 more
europepmc   +1 more source

Safety and Efficacy of Nusinersen Focusing on Renal and Hematological Parameters in Spinal Muscular Atrophy. [PDF]

Brain Behav
Bahadır Şenol H, Yıldız G, Polat Aİ, Aydın A, Hız AS, Soylu A, Yiş U.   +6 more
europepmc   +1 more source

Towards a Definition of Physiologic Vulnerability in Pediatric Spine Surgery: Identification of Key Risk Factors in a Cohort Study of Children With Neuromuscular Disease Undergoing Spinal Fusion. [PDF]

Global Spine J
Ghauri MS, Rajkumar S, Stone LE, Kelly MP, Iyer RR, Bauer J, Ames CP, Newton PO, Gonda DD, Levy ML, Ravindra VM.   +10 more
europepmc   +1 more source

Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands. [PDF]

Eur J Health Econ
van der Schans S, Velikanova R, Weidlich D, Howells R, Patel A, Bischof M, Postma MJ, Boersma C.   +7 more
europepmc   +1 more source