Results 171 to 180 of about 45,438 (256)

Bridging Kidney Organoid Innovation and Regenerative Medicine: Current Advances and Future Directions

Cell Proliferation, EarlyView.
This review outlines kidney organoid differentiation strategies, key developmental mechanisms, and engineering and multi‐omics approaches driving their progress toward regenerative medicine. ABSTRACT Chronic kidney disease (CKD) has emerged as a critical public health challenge worldwide, and organ donor shortages underscore the urgent need for ...
Bohong Guo, Jingyuan Zhang, Xuening Fang, Yangyang Liu, Yueyang Deng, Yuxin Xue, Zhengxi Wang, Xiaotao Dong, Mengqi Jia, Xiaodong Li   +9 more
wiley   +1 more source

Evaluation of the Telomere Length in Patients with Spinal Muscular Atrophy. [PDF]

Int J Mol Sci
Okur Altındaş B, Öktem S, Çarman KB, Yıldırım MS.   +3 more
europepmc   +1 more source

Autism spectrum disorder in children with spinal muscular atrophy type 1: Case series

Developmental Medicine &Child Neurology, EarlyView.
Autism spectrum disorder (ASD) was identified in 37.5% of children with SMA (n = 13). While IQ did not differ significantly between groups, adaptive functioning was reduced in those with co‐occurring ASD. Results support routine early neurodevelopmental screening in children with spinal muscular atrophy.
Lorena V. Rezende, Adriana B. Ortega, Daniel A. Do Valle, Tiago S. Bara, Mara L. Cordeiro   +4 more
wiley   +1 more source

Nanotube-Assisted Motor Neuron and Neuromuscular Junction Stabilization in Spinal Muscular Atrophy: A Hypothesis for Adjunctive Therapy. [PDF]

Neurol Int
Fajkić A, Belančić A, Pilipović K, Rački V, Mežnarić S, Janković T, Gkrinia EMM, Vitezić D, Mršić-Pelčić J.   +8 more
europepmc   +1 more source

Ethics and equity in access to disease‐modifying therapies and newborn screening for spinal muscular atrophy: A scoping review

Developmental Medicine &Child Neurology, EarlyView.
Outcomes of children with cerebral palsy receiving long‐term respiratory support. Aim To review barriers to ethical and equitable access to disease‐modifying therapies (DMTs) and newborn screening (NBS) for spinal muscular atrophy (SMA). Method We searched PubMed, Scopus, Web of Science, EBSCOhost, the Cochrane Library, Google Scholar, and Primo for ...
Serini Murugasen, Mariana Kruger, Marc Blockman, Jo M. Wilmshurst   +3 more
wiley   +1 more source

Real-world evidence of Nusinersen treatment for patients with spinal muscular atrophy in the Kingdom of Saudi Arabia: Initial insights from the Saudi national spinal muscular atrophy program. [PDF]

Saudi Med J
Al-Jedai AH, Almudaiheem HY, Alaama TY, Alsaman ASS, Abdo Bashiri F, Almuhaizae MA, Alghamdi FA, Alsakran AA, Matter EAY, Ibrahim FZ.   +9 more
europepmc   +1 more source

Performance outcomes of the PEDI‐CAT for assessing functional ability in the population with leukodystrophy

Developmental Medicine &Child Neurology, EarlyView.
Abstract Aim To describe the use of the Pediatric Evaluation of Disability Index‐Computer Adapted Test (PEDI‐CAT), a parent‐reported outcome, and determine functional performance in a cohort with leukodystrophy. Method This was a cross‐sectional observational study.
Stacy V. Cusack, Allan M. Glanzman, Francesco Gavazzi, Sarah Woidill, Abbas F. Jawad, Timothy Estilow, Amy T. Waldman, Adeline Vanderver, Laura Adang, Children's Hospital of Philadelphia Leukodystrophy Workgroup, Todd Levy, Michelle Fitzgerald, Ann T. Harrington, Emma McKenzie, Elizabeth Balance, Tracy Kornafel, Kaleb Kelley, Peter Hong, Sarah Pham, Aaliyah White, Miriam Lerner   +20 more
wiley   +1 more source

Haloperidol induces neuroprotection and enhances neuromuscular function in both murine and human models of spinal muscular atrophy. [PDF]

Exp Mol Med
Menduti G, Perez-Gomez R, Berenger-Currias N, Ruatti C, Espinosa-Espinosa J, Januel C, Konieczny P, Artero R, Martinat C, Boido M.   +9 more
europepmc   +1 more source

The Spinal Muscular Atrophy Functional Classification System

Developmental Medicine &Child Neurology, EarlyView.
The Spinal Muscular Atrophy Functional Classification System (SMAFCS) is a new four‐level functional mobility classification for spinal muscular atrophy, derived from Hammersmith Functional Motor Scale Expanded (HFMSE) thresholds and Functional Mobility Scale (FMS) groupings.
Jason J. Howard, H. Kerr Graham, M. Wade Shrader, Mary Schroth, Manish Kumar, Sadettin Ciftci   +5 more
wiley   +1 more source