Results 151 to 160 of about 1,822 (274)

Single-dose GC101 gene therapy for spinal muscular atrophy types II and III: an open-label single-arm study. [PDF]

World J Pediatr
Ma XW, Jiang XY, Li ZQ, Dong XY, Ma WH, Wang YX, Yu SQ, Gu RJ, Wang XD, Zhong BM, He F, Xu J, Mao YY, Zhang YP, Zhang S, Li T, Li CY, Zhang S, Yang X, Zhu LN, Wu ZJ, Zhou B, Zhuang L, Li QP, Wu XB, Feng ZC.   +25 more
europepmc   +1 more source

Vitamin D Deficiency as a Debatable Modulator of Outcomes in Transfusion‐Dependent Thalassemia; An Overview of the Latest Findings: A Narrative Review

Health Science Reports, Volume 8, Issue 10, October 2025.
ABSTRACT Background and Aims Vitamin D deficiency (VDD) is highly prevalent in transfusion‐dependent thalassemia (TDT), yet its clinical implications beyond bone health remain controversial. This review explores the risk factors and clinical outcomes of VDD in TDT, with particular focus on its potential roles in cardiac iron deposition, osteoporosis ...
Ali Bazi, Mahdieh Poodineh Moghadam, Jafar Baranipour, Hajar Noori Sanchooli, Hamed Soleimani Samarkhazan, Omolbanin Sargazi Aval, Mojtaba Aghaei   +6 more
wiley   +1 more source

The effect of thoracolumbosacral orthosis on scoliosis progression and chest deformity in children with type 1 spinal muscular atrophy: A randomized controlled trial. [PDF]

PLoS One
Dansuk E, Van Den Berg ANT, Ata G, Yıkılmaz SK, Oktem S.   +4 more
europepmc   +1 more source

Frequency and satisfaction of conventional and complementary or alternative therapies for neuromuscular disorders. [PDF]

Neurol Res Pract, 2023
Böhne LÄ, Wirner C, Schoser B, Schröter C, Baum P.   +4 more
europepmc   +1 more source

MyomiRs Expression in Limb Girdle Muscular Dystrophy

IUBMB Life, Volume 77, Issue 10, October 2025.
ABSTRACT This manuscript is a comprehensive review focused on the role of microRNAs (miRs)—short RNA molecules—in Limb Girdle Muscular Dystrophy (LGMD). LGMD encompasses various and heterogeneous rare genetic neuromuscular diseases, characterized by the progressive wasting and deterioration of muscle fibers, predominantly affecting the pelvic and ...
G. Breveglieri, M. T. Altieri, M. T. Rodia, R. Costa, F. Frabetti, G. Cenacchi, G. Sabbioni, M. Borgatti   +7 more
wiley   +1 more source

Postural Tremor Caused by Hirayama Disease Mimicking Essential Tremor. [PDF]

Tremor Other Hyperkinet Mov (N Y)
Xie N, Sun Q, Yang G.
europepmc   +1 more source

Exploring the 3D Bioprinting Landscape in the Delivery of Active Pharmaceutical Compounds for Therapeutic and Regenerative Medicine Applications

Journal of Biomedical Materials Research Part B: Applied Biomaterials, Volume 113, Issue 10, October 2025.
Three‐dimensional (3D) bioprinting is revolutionizing drug delivery and regenerative medicine by enabling personalized, controlled‐release therapeutic systems. This review explores advancements in biocompatible bioinks, nanomedicines, and 3D‐printed drug delivery constructs, including scaffolds, implants, and bioactive hydrogels.
Khonzisizwe Somandi, Yahya E. Choonara
wiley   +1 more source

A perioperative nursing care protocol for patients with spinal muscular atrophy (SMA) type II or type III undergoing spinal surgery: a 4-year experience in 24 patients. [PDF]

Orphanet J Rare Dis
Li G, Xu K, Liu D, Wu N, Zhang TJ, Chen Y.   +5 more
europepmc   +1 more source

The Role of Healthy Relationships and Social Connectivity in the Mediterranean Lifestyle: An Umbrella Review of Reviews

Lifestyle Medicine, Volume 6, Issue 4, October 2025.
ABSTRACT Introduction Social connectivity and interpersonal relationships underpin all aspects of lifestyle. As part of a broader research collaboration on the Mediterranean lifestyle, we examined existing evidence on social connectivity patterns in Mediterranean regions and how they differed from those in non‐Mediterranean regions to explore the role ...
Gulnur Yaman‐Dent, Gloria Dent, Rob Lawson, Hannah Maycroft, Catalina Figueroa, Linda Erringto, Nicola O'Brien, Emily Symington, Beth Frates, Gregory Maniatopoulos, Ann Johnson, Aleksandra Telinga, Fraser Birrell   +12 more
wiley   +1 more source

A contemporary analysis of the Australian clinical and genetic landscape of spinal muscular atrophy: a registry based study. [PDF]

Lancet Reg Health West Pac
Balaji L, Forbes R, Cairns A, Sampaio H, Kornberg AJ, Sanders L, Lamont P, Liang C, Jones KJ, Nowak K, O'Gorman C, Woodcock I, Briggs N, Yiu EM, Farrar MA, Kariyawasam D.   +15 more
europepmc   +1 more source