Results 161 to 170 of about 1,822 (274)

Neuroleukemiosis Masquerading as Drug Toxicity in an Adolescent With Refractory AML

American Journal of Hematology, Volume 100, Issue 10, Page 1853-1858, October 2025.
Nia Choi, Deborah Schady, Tim Lotze, Jill Ann Jarrell, Alexandra Rodriguez‐Hernandez, Sandra Aziz, Karen K. Moeller, Joanna S. Yi, Suzanne Woodbury, Andrea N. Marcogliese, Zoann E. Dreyer, Eric S. Schafer   +11 more
wiley   +1 more source

Gene and RNA Editing: Revolutionary Approaches to Treating Diseases

MedComm, Volume 6, Issue 10, October 2025.
The image illustrates gene editing technologies: DNA editing using CRISPR–Cas9 and RNA editing via Cas13d, with their clinical applications and ethical risks. DNA editing allows precise gene modifications for conditions like amyotrophic lateral sclerosis [ALS] and Huntington's disease, while RNA editing supports multiplexed modifications.
Jia‐Mei Li, Jie Huang, Yan Liao, Ting Hu, Chang‐Li Wang, Wang‐Zheqi Zhang, Chen‐Wei Huang   +6 more
wiley   +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

Sensory dysfunction in SMA type 2 and 3 - adaptive mechanism or concomitant target of damage? [PDF]

Orphanet J Rare Dis
Koszewicz M, Ubysz J, Dziadkowiak E, Wieczorek M, Budrewicz S.   +4 more
europepmc   +1 more source

Motor Unit Number Index (MUNIX) in Control Children: Reference Values and Reliability

Muscle &Nerve, Volume 72, Issue 4, Page 625-631, October 2025.
ABSTRACT Introduction/Aims The motor unit number index (MUNIX) is recognized as a reliable electrophysiological biomarker, and reference values are available for healthy adults but not for children. The aim of this study was to determine reference MUNIX values in healthy children.
Christophe Boulay, Emilien Delmont, Frédérique Audic, Cécile Halbert, Sébastien Pesenti, Brigitte Chabrol, Béatrice Desnous, Shahram Attarian   +7 more
wiley   +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

Clinical Utility of Far‐Field Potentials in Amyotrophic Lateral Sclerosis

Muscle &Nerve, Volume 72, Issue 4, Page 616-624, October 2025.
ABSTRACT Introduction/Aims Far field potentials (FFP) have been proposed as a reliable neurophysiological prognostic biomarker in amyotrophic lateral sclerosis (ALS). This study evaluates the diagnostic utility of ulnar nerve FFP in ALS. Methods Comprehensive peripheral neurophysiological assessments were conducted in 62 ALS and 43 ALS‐mimicking ...
Aicee Dawn Calma, Nathan Pavey, Claudia Santos Silva, Yukiko Tsuji, Mehdi A. J. Van den Bos, Michelle A. Farrar, Parvathi Menon, Steve Vucic   +7 more
wiley   +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

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

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