Results 171 to 180 of about 121,613 (384)

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +1 more source

Antisense Oligonucleotides Shed New Light on the Pathogenesis and Treatment of Spinal Muscular Atrophy [PDF]

, 2012
Jiing‐Kuan Yee, Ren-Jang Lin
openalex   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

Biomaterial design strategies for enhancing mitochondrial transplantation therapy

BMEMat, EarlyView.
Biomaterials to facilitate mitochondrial transplantation therapy: biomaterials as barriers to protect mitochondria from pathophysiological microenvironments, like osmotic stress caused by the excessive concentration of calcium ion, reactive oxygen species, and advanced glycation end products; biomaterials integrating with biochemical cues to improve ...
Shaoyang Kang, Yushan Zhang, Hang Li, Sirui Peng, Donghao Lyu, Chunxiao Zhou, Sheng Ding, Zujian Feng, Pingsheng Huang, Chuangnian Zhang, Hongjun Wang, Deling Kong, Weiwei Wang   +12 more
wiley   +1 more source

Motor fiber function in spinal muscular atrophy—analysis of conduction velocity distribution [PDF]

, 2023
Magdalena Koszewicz, Jakub Ubysz, Edyta Dziadkowiak, Małgorzata Wieczorek, Sławomir Budrewićz   +4 more
openalex   +1 more source

Nanomaterial‐based immune therapeutic strategies in neurodegenerative diseases

BMEMat, EarlyView.
This review highlights the immunomodulatory potential of nanomaterials (NMs) in treating neurodegenerative diseases (NDs). It focuses on their roles in regulating innate and adaptive immune responses to maintain immune homeostasis. By providing insights into these mechanisms, the review lays the groundwork for innovative NMs therapeutic strategies to ...
Xinru Zhou, Jia Liu, Shulin Lai, Suhan Yin, Bingmin Luo, Longquan Shao, Yiyuan Kang   +6 more
wiley   +1 more source

Survival Motor Neuron (SMN) protein is required for normal mouse liver development [PDF]

, 2016
Spinal Muscular Atrophy (SMA) is caused by mutation or deletion of the survival motor neuron 1 (SMN1) gene. Decreased levels of, cell-ubiquitous, SMN protein is associated with a range of systemic pathologies reported in severe patients.
Francesco, M, Gillingwater, TH, Maxwell, GK, Parson, SH, Powis, RA, Szunyogova, E, Zhou, H   +6 more
core  

Beyond mammals: the evolution of chewing and other forms of oropharyngeal food processing in vertebrates

Biological Reviews, EarlyView.
ABSTRACT Oropharyngeal food processing exhibits a remarkable diversity among vertebrates, reflecting the evolution of specialised ‘processing centres’ associated with the mandibular, hyoid, and branchial arches. Although studies have detailed various food‐processing strategies and mechanisms across vertebrates, a coherent and comprehensive terminology ...
Daniel Schwarz, Maja Mielke, Stephan Handschuh, Anthony Herrel, Patrick Lemell, Léa Da Cunha, Nicolai Konow   +6 more
wiley   +1 more source

A Core Head, Neck, and Neuroanatomy Syllabus for Physical Therapy Student Education

Clinical Anatomy, EarlyView.
ABSTRACT Head, neck, and neuroanatomy are essential components of physical therapy education due to their broad clinical applications. Detailed syllabi exist for medical students, yet none have been developed for physical therapy. This study aimed to produce an International Federation of Associations of Anatomists core head, neck, and neuroanatomy ...
Stephanie J. Woodley, Brooke Willoughby, Alexandra L. Webb, Natasha A. M. S. Flack, Laura Y. Whitburn   +4 more
wiley   +1 more source

Reduced mesencephalic astrocyte–derived neurotrophic factor expression by mutant androgen receptor contributes to neurodegeneration in a model of spinal and bulbar muscular atrophy pathology

Neural Regeneration Research
Spinal and bulbar muscular atrophy is a neurodegenerative disease caused by extended CAG trinucleotide repeats in the androgen receptor gene, which encodes a ligand-dependent transcription factor.
Yiyang Qin, Wenzhen Zhu, Tingting Guo, Yiran Zhang, Tingting Xing, Peng Yin, Shihua Li, Xiao-Jiang Li, Su Yang   +8 more
doaj   +1 more source