Results 81 to 90 of about 12,163 (214)

Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito, Kamal Benguerba, Ken Tsuchida, Kazushige Yazawa, Isao Tsumiyama, Hiromitsu Kayama, Sandra P. Reyna, Farid Khan, Richard S. Finkel   +8 more
wiley   +1 more source

Digital Cognitive Phenotyping for Differential Diagnosis and Monitoring in Neurological Conditions

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To assess the utility, accessibility, and equivalence to supervised scales of online cognitive assessment in older individuals with cognitive impairment. Methods Patients with Alzheimer's disease (AD, n = 31), idiopathic normal pressure hydrocephalus (iNPH, n = 26), and traumatic brain injury (TBI, n = 23) completed online cognitive ...
Martina Del Giovane, Valentina Giunchiglia, Michael C. B. David, Magdalena A. Kolanko, William R. Trender, Peter J. Hellyer, Harmeena Kaur, David J. Sharp, Christopher Carswell, Paresh A. Malhotra, Adam Hampshire   +10 more
wiley   +1 more source

Rheology and Infrared Spectrum of Diatomite Asphalt Concrete

Kuangchan zonghe liyong
This is an article in the field of ceramics and composites. In order to study the influence of diatomite content on the physical properties, rheological properties and road performance of asphalt mixtures, the basic physical tests, rheological tests ...
Yi XU, Yin ZHANG
doaj   +1 more source

Dynamic Stability of Rotating Blades with Transverse Cracks

Shock and Vibration, 2003
In this paper, the main objective is to examine the effects of transverse cracks on the dynamic instability regions of an axially loaded rotating blade. The blade is modeled as an Euler-Bernoulli beam.
T.Y. Ng, K.Y. Lam, Hua Li
doaj   +1 more source

Progressive Parkinsonism in PPP2R5D‐Related Neurodevelopmental Disorder

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT PPP2R5D‐related neurodevelopmental disorder (Houge–Janssens syndrome type 1) is a rare autosomal dominant condition characterized by macrocephaly, intellectual disability, and epilepsy. Progressive parkinsonism is an emerging adult phenotype that neurologists should be aware of since timely genetic diagnosis opens the door to disease‐modifying
Katerina Bernardi, Enrique Gonzalez Saez‐Diez, Joshua Rong, Beril Ay, Shabbir Merchant, Kathryn Yang, Darius Ebrahimi‐Fakhari   +6 more
wiley   +1 more source

Aluminate Long Afterglow Luminescent Materials in Road Marking Field Research Progress and Development: A Review

Buildings
This paper reviews the research progress and development of aluminate long afterglow luminescent materials in the field of road marking, especially the study of rare earth ion-activated strontium aluminate (SrAl2O4: Eu2+, Dy3+)-based long afterglow ...
Fangzhi Zhang, Yue Xie, Xiaokang Zhao, Yinzhang He, Jianzhong Pei, Yuanhe Xing, Shaobo Wang, Jiupeng Zhang   +7 more
doaj   +1 more source

Re‐Purposing Sapropterin (Kuvan) for ACTA2‐Related Multisystemic Smooth Muscle Dysfunction Syndrome: A Translational Mechanistic and First‐In‐Human Therapeutic Report

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Multisystemic smooth muscle dysfunction syndrome (MSMDS) is an ultra‐rare, ACTA2‐related disorder characterized by severe cerebrovascular disease, aortic aneurysms, and smooth muscle dysfunction. Using molecular dynamics simulations and in silico drug screening, we identified that sapropterin dihydrochloride (Kuvan) is a candidate capable of ...
Moran Hausman‐Kedem, Noa Bielopolski, Vijai Krishnan, Shane Wald‐Altman, Shelly I Shrian, Omer Bar‐Yosef, Livia Kapusta, Claire Shamber, Patricia L. Musolino, Miguel Weil   +9 more
wiley   +1 more source

Phase-Change/Salt-Based Slow-Release Composite Material for Anti-Icing and Snow-Melting

Buildings
Currently, self-desiccating asphalt mixtures on roads mainly incorporate phase-change materials or salt-based slow-release agents individually for de-icing. However, pure phase-change material mixtures have limited anti-freezing efficiency and short heat-
Chuanshan Wu, Dongxing Gao, Haonan Shangguan, Renshan Chen, Changlin Hou   +4 more
doaj   +1 more source

Posterior Disconnection Syndrome in Early‐Stage Adult‐Onset Cerebral Adrenoleukodystrophy

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Adult‐onset cerebral adrenoleukodystrophy is potentially treatable but often difficult to recognize before advanced cerebral involvement. Herein, we describe three men with early‐stage disease who initially presented with subtle visual complaints rather than subcortical dementia. Targeted neuropsychological testing revealed higher‐order visual
Kazuto Katsuse, Takashi Matsukawa, Yumiko Nakamura, Kazuo Kakinuma, Shoko Ota, Ryo Hara, Saki Nakashima, Kaoru Ito, Yutaro Nakamori, Toshiyuki Kakumoto, Masashi Hamada, Shigenori Kanno, Kyoko Suzuki, Tatsushi Toda   +13 more
wiley   +1 more source

High Health Care Utilization Preceding Diagnosis With Juvenile Idiopathic Arthritis

Arthritis Care &Research, EarlyView.
Objective Although early diagnosis improves long‐term outcomes, patients with juvenile idiopathic arthritis (JIA) often experience prolonged, circuitous paths to diagnosis. To inform diagnostic improvement, we sought to characterize health care utilization in the year preceding diagnosis. Methods We identified 10,021 patients with an incident diagnosis
Anna Costello, Rui Xiao, Xuemei Zhang, Jahan Jazayeri, Irit R. Rasooly, Pamela F. Weiss   +5 more
wiley   +1 more source