Results 171 to 180 of about 1,822 (274)

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

Risdiplam in Adult Patients With 5q Spinal Muscular Atrophy: A Single-Center Longitudinal Study. [PDF]

Muscle Nerve
Gavriilaki M, Moschou M, Pagiantza M, Arnaoutoglou M, Kimiskidis V.   +4 more
europepmc   +1 more source

Parental perspectives on family‐centered care in pediatric neurology: An explanatory sequential mixed‐methods study

Developmental Medicine &Child Neurology, Volume 67, Issue 10, Page 1340-1353, October 2025.
The proposed conceptual framework highlighting parents' central role in their child's healthcare experience. This original article is commented by Kettle‐Frisby on pages 1247–1248 of this issue. Abstract Aim To explore parents' experiences of family‐centered care (FCC) in a pediatric neurology clinic. Method In this explanatory sequential mixed‐methods
Ege Sarikaya, Courtney B. Cook, Kathryn A. Selby, Ye Shen, GenCOUNSEL Study, Alison M. Elliott, Jehannine Austin, Bartha Knoppers, Larry D. Lynd, Alivia Dey, Shelin Adam, Nick Bansback, Patricia Birch, Lorne Clarke, Nick Dragojlovic, Jan Friedman, Debby Lambert, Daryl Pullman, Alice Virani, Wyeth Wasserman, Ma’n Zawati, Alison M. Elliott   +21 more
wiley   +1 more source

Administration practices of and adherence to nusinersen in children with spinal muscular atrophy: a multicenter disease registry study in China. [PDF]

BMC Pediatr
Peng J, Yao X, Luo R, Wang X, Wu L, Zhong J, Jin R, Lu X, Liang J, Hong S, Yang L, Zhang X, Mao S, Tao Z, Hu J, Sun D, Wang H, Zhang L, Xia Y, Chen K, Wang Y.   +20 more
europepmc   +1 more source

2025 ACVIM Forum Research Abstract Program

Journal of Veterinary Internal Medicine, Volume 39, Issue 6, November/December 2025.
wiley   +1 more source

Families' Perceptions of Powered Mobility for Participation in Children With Spinal Muscular Atrophy Type 1: A Photovoice Study. [PDF]

Health Expect
Coello-Villalón M, Díaz-López CI, López-Muñoz P, Romay-Barrero H, Pacheco-da-Costa S, Plasencia-Robledo M, Longo E, Palomo-Carrión R.   +7 more
europepmc   +1 more source

Sleep Medicine—What's in a Name?

Journal of Sleep Research, Volume 34, Issue 5, October 2025.
ABSTRACT Sleep medicine has matured into a recognised medical discipline, characterised by defined diagnostic concepts, evidence‐based treatments, and significant progress in understanding sleep physiology and disorders. Sleep and its disturbances impact virtually every aspect of health and well‐being.
Dirk A. Pevernagie, Erna Sif Arnardottir, Oliviero Bruni, Sarah Hartley, Gert Jan Lammers, Tiina Paunio, Dieter Riemann, Renata L. Riha   +7 more
wiley   +1 more source

Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy. [PDF]

Gene Ther
Pongsakornkullachart P, Kulsirichawaroj P, Kongkasuwan R, Tovichien P, Jitwongwai S, Kanjanauthai S, Preeprem N, Limpaninlachat S, Sermpon N, Sanmaneechai O.   +9 more
europepmc   +1 more source

Reduced white matter integrity and disrupted brain network in children with type 2 and 3 spinal muscular atrophy. [PDF]

J Neurodev Disord
Nie H, Lan S, Wang H, Xiang P, Yan M, Fan Y, Shen W, Li Y, Tang W, Yang Z, Liang Y, Chen Y.   +11 more
europepmc   +1 more source

Clinical Phenotype and Neuroimaging Findings in Siblings with COX15 Deficiency: Case Report and Review of Previously Reported Cases

Movement Disorders Clinical Practice, Volume 12, Issue 10, Page 1642-1646, October 2025.
Haya S. AlFaris, Sangeetha Yoganathan, Marcus N. Callister, Liali Aljouda, Vivek Pai, Pradeep Krishnan, Andrea LeBlanc‐Millar, Christos Ganos, Carolina Gorodetsky   +8 more
wiley   +1 more source