Results 61 to 70 of about 1,822 (274)

Recent Utilization of Pediatric Extrapolation and Modeling and Simulation Approaches in Pediatric Drug Development in Japan

The Journal of Clinical Pharmacology, EarlyView.
Abstract In Japan, the percentage of approved drugs with pediatric indications increased to 30% in 2010‐2015, but no further increase was observed through 2020. The Ministry of Health, Labor, and Welfare in Japan presented draft future directions to promote pediatric drug development, where the modeling and simulation (M&S) approach was introduced as a
Akinori Nakashima, Akihide Tsujimoto, Masako Saito, Jun Takeda, Makiko Natori, Satoshi Shoji   +5 more
wiley   +1 more source

Spinal muscular atrophy [PDF]

, 1995
Spinal Muscular Atrophy (SMA) is one of many neuromuscular diseases affect ing motor neurons and skeletal muscles. This disorder causes deterioration of the motor neurons (specifically the Anterior Horn Cells of the spinal cord). These motor neurons that
Nowak, Deborah
core   +1 more source

An examination of factors affecting the efficacy of physiotherapeutic scoliosis‐specific exercises in the management of adolescent idiopathic scoliosis

Journal of Intelligent Medicine, EarlyView.
We apologize for the omission. Please find attached the Graphical Abstract we have prepared, which visually summarizes the key elements of our study, including adolescent idiopathic scoliosis, physiotherapeutic scoliosis‐specific exercises (PSSE), influencing factors (initial Cobb angle, skeletal maturity, exercise compliance, and vertebral rotation ...
Lu Liu, Wenjie Liu, Ding Xiang, Jing Wang, Xin Li, Jiaxin Dong, Haisha Xia, Xizi Song   +7 more
wiley   +1 more source

Discovery of a small molecule probe that post-translationally stabilizes the survival motor neuron protein for the treatment of spinal muscular atrophy. [PDF]

, 2017
Spinal muscular atrophy (SMA) is the leading genetic cause of infant death. We previously developed a high-throughput assay that employs an SMN2-luciferase reporter allowing identification of compounds that act transcriptionally, enhance exon recognition,
Androphy, Elliot J., Boaler, Patrick J., Burnett, Barrington G., Calder, Alyssa N., Cherry, Jonathan J., Choi, Sungwoon, Cuny, Gregory D., Fritsche, Melanie, Glicksman, Marcie A., Hodgetts, Kevin J., Kern, Nicholas L., Li, Hongxia, Lorson, Christian L., Lusic, Hrvoje, Quist, Kevin M., Rietz, Anne, Xing, Xuechao   +16 more
core   +2 more sources

Central Pathophysiology and Brain Network Changes Related to Camptocormia in Parkinson's Disease

Movement Disorders, EarlyView.
ABSTRACT Background Studies on brain connectivity offer important insights into the changes that occur in central network diseases such as Parkinson's disease (PD). Camptocormia, a condition characterized by abnormal flexion of the trunk, often occurs in advanced PD, but its underlying mechanisms are not yet clear.
Tauqeer Anjum, Jan Raethjen, Hao Ding, Nabin Koirala, Rüdiger Pryss, Robin Wolke, Chi Wang Ip, Günther Deuschl, Jens Volkmann, Nils G. Margraf, Muthuraman Muthuraman   +10 more
wiley   +1 more source

Perioperative rehabilitation management for a child with spinal muscular atrophy: one case report

Chinese Journal of Contemporary Neurology and Neurosurgery, 2023
LI Wen‑zhu, WANG Chen‑hao, HUANG Zhen, WEI Cui‑jie, XIONG Hui, XU Bei‑yu, LI Chun‑de, QUE Cheng‑li   +7 more
doaj   +1 more source

Analysis of a Modified Version of the Inventory of Non‐Ataxia Signs Over 12 Years in Patients with Friedreich's Ataxia in the EFACTS Study

Movement Disorders, EarlyView.
Abstract Background Friedreich's ataxia is a rare, neurodegenerative, multisystem disorder. While ataxia is a hallmark, non‐ataxia signs, including muscle weakness, spasticity, and dysphagia are equally disabling. The Inventory of Non‐Ataxia Signs (INAS) is a symptom list transformable to a 16‐item count.
Stella Andrea Lischewski, Imis Dogan, Paola Giunti, Michael H. Parkinson, Caterina Mariotti, Alexandra Durr, Claire Ewenczyk, Sylvia Boesch, Wolfgang Nachbauer, Thomas Klopstock, Claudia Stendel, Francisco Javier Rodríguez de Rivera Garrido, Ludger Schöls, Zofia Fleszar, Thomas Klockgether, Marcus Grobe‐Einsler, Ilaria Giordano, Myriam Rai, Massimo Pandolfo, Heike Jacobi, Ralf‐Dieter Hilgers, Jörg B. Schulz, Kathrin Reetz, on behalf of the EFACTS Study Group   +23 more
wiley   +1 more source

Exploring Autophagy Inducing Molecules: Targeting Diverse Pathways in Alzheimer's Disease Management

Medicinal Research Reviews, EarlyView.
ABSTRACT Neurodegenerative disorders, including Alzheimer's disease (AD), impose a significant burden on society due to their progressive nature and the associated healthcare costs. Autophagy, a vital cellular degradation process, has emerged as a promising therapeutic target in these disorders.
Baljinder Singh, Shubham Mahajan, Sadikalmahdi Abdella, Rehan Khan, Sanjay Garg   +4 more
wiley   +1 more source

Feasibility of a Home‐Based Exergaming Intervention for Youth With Spinal Muscular Atrophy

Muscle &Nerve, EarlyView.
ABSTRACT Introduction/Aims Approaches to optimize physical activity in youth with spinal muscular atrophy (SMA) are rapidly evolving. The primary objective of this study was to assess the feasibility of a fit‐for‐purpose home‐based exergaming intervention in children and youth with SMA and peer controls.
Ihsane Iraqi, Pamela Ng, Xing Chen, Niamh Cushen, Juergen Gottowik, David Herzig, Shaainthabie Karthigesu, Danielle Levac, Alex MacKenzie, Jean K. Mah, Slawomir Opalka, Beth Potter, Kathryn Selby, Jordan Sheriko, Maureen Smith, Sarah Turgeon‐Desilets, Angelina Woof, Maryam Oskoui   +17 more
wiley   +1 more source

Study of survival of motor neuron (SMN) and neuronal apoptosis inhibitory protein (NAIP) gene deletions in SMA patients [PDF]

, 2005
In view of the paucity of deletion studies of survival of motor neuron (SMN) and neuronal apoptosis inhibitor protein (NAIP) genes in Indian SMA patients, this study has been undertaken to determine the status of SMN1, SMN2 and NAIP gene deletions in ...
Kalita, J., Kesari, A., Mishra, V. N., Misra, U. K., Mittal, B., Patil, S. J., Phadke, S. R., Pradhan, S.   +7 more
core   +1 more source