Results 171 to 180 of about 9,396,775 (321)

Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT The placebo response in clinical trials in ataxias complicates outcome interpretation and potentially obscures genuine treatment effects. We analyzed placebo group data from past trials in Friedreich Ataxia and observed notable responses in appendicular items, in contrast to minimal changes in axial function, as measured by respective ...
Christian Rummey, Jennifer M. Farmer, David R. Lynch   +2 more
wiley   +1 more source

Patient and Clinician Perspectives on Pulmonary Arterial Hypertension Clinical Trial Endpoints - A Qualitative Study [PDF]

, 2021
R. Goodman, T. Lapatra, Nadine Al‐Naamani, S.M. Kawut, Katherine R. Courtright   +4 more
openalex   +1 more source

Basilar Artery Occlusion Stroke Managed With Tenecteplase Versus Alteplase Before Endovascular Treatment (BAO‐TNK)

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective To compare the effectiveness and safety of tenecteplase (TNK) versus alteplase (TPA) in patients with basilar artery occlusion prior to endovascular treatment (EVT). Methods In this retrospective multicenter study (BAO‐TNK), we analyzed consecutive BAO patients from 14 U.S.
Rahul R. Karamchandani, Dylan N. Wolman, Tyler M. Bielinski, Nitin Goyal, Jeremy B. Rhoten, Mahesh V. Jayaraman, Christian T. Sidebottom, Bradley A. Gross, Alhamza R. Al‐Bayati, Mohamed F. Doheim, Matthew K. Tobin, Anoop Chinthala, Avi Gajjar, Justin Mascitelli, Matthew R. Webb, Jan‐Karl Burkhardt, Kyle W. Scott, Visish Srinivasan, Daniel Tonetti, Manisha Koneru, David J. Altschul, Dhrumhil Vaishnav, Sumeet Multani, Thana Theofanis, William J. Ares, Je Yeong Sone, Ramin Zand, Sasan Bahrami, Jiang Li, Gary Defilipp, Dale Strong, Radmehr Torabi, Krisztina Moldovan, Kelsey E. Kline, Clemens M. Schirmer, Raul G. Nogueira, Alexandra R. Paul, Bradley N. Bohnstedt, Philipp Hendrix   +38 more
wiley   +1 more source

Health‐Related Quality of Life in Rare Forms of Childhood‐Onset Hereditary Spastic Paraplegia

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT We assessed health‐related quality of life (HRQoL) in 80 children with rare hereditary spastic paraplegias using the Caregiver Priorities and Child Health Index of Life with Disabilities and clinician‐reported outcomes. HRQoL was consistently reduced, particularly in relation to motor, autonomic, and bulbar symptoms.
Henri J. D. Schmidt, Nicole Battaglia, Joshua Rong, Amy Tam, Siofra Carty, Vicente Quiroz, Kathryn Yang, Zainab Zaman, Luca Schierbaum, Katerina Bernardi, Julian E. Alecu, Darius Ebrahimi‐Fakhari   +11 more
wiley   +1 more source

Physical Activity in Pediatric Pulmonary Arterial Hypertension Measured by Accelerometry. A Candidate Clinical Endpoint

American Journal of Respiratory and Critical Care Medicine, 2017
Willemijn M. H. Zijlstra, Mark-Jan Ploegstra, Theresia R. Vissia-Kazemier, M. Roofthooft, Gideon J. du Marchie Sarvaas, B. Bartelds, A. Rackowitz, F. van den Heuvel, H. Hillege, G. Plasqui, R. Berger   +10 more
semanticscholar   +1 more source

Chronological and Spatial Distribution of Skeletal Muscle Fat Replacement in FHL1‐Related Myopathies

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objectives Variants in the FHL1 gene cause FHL1‐related myopathies (FHL1‐RMs), a group of neuromuscular disorders with diverse clinical presentations. This study aimed to comprehensively characterize the spatial and temporal patterns of skeletal muscle fat replacement throughout the whole body in FHL1‐RMs, to examine disease progression over ...
Rui Shimazaki, Satoru Noguchi, Hotake Takizawa, Yasushi Oya, Yuji Takahashi, Hirofumi Komaki, Hajime Arahata, Shinichiro Hayashi, Ichizo Nishino   +8 more
wiley   +1 more source

Forced Expiratory Flow (FEF_25-75%) as a Clinical Endpoint in Children and Adolescents with Symptomatic Asthma Receiving Tiotropium: A Post Hoc Analysis. [PDF]

Pulm Ther, 2020
Szefler SJ, Goldstein S, Vogelberg C, Bensch GW, Given J, Jugovic B, Engel M, Moroni-Zentgraf PM, Sigmund R, Hamelmann EH.   +9 more
europepmc   +1 more source

Supplementary Data from Statistical Considerations for Analyses of Time-To-Event Endpoints in Oncology Clinical Trials: Illustrations with CAR-T Immunotherapy Studies

, 2023
Yimei Li, Wei‐Ting Hwang, Shannon L. Maude, David T. Teachey, Noelle V. Frey, Regina M. Myers, Allison Barz Leahy, Hongyan Liu, David L. Porter, Stephan A. Grupp, Pamela A. Shaw   +10 more
openalex   +1 more source

Reduction in Renal Relapse and Preservation of Long‐Term Kidney Function After Lupus Low Disease Activity in Patients With Lupus Nephritis

Arthritis Care &Research, EarlyView.
Objective Lupus low disease activity state (LLDAS) is a validated treatment target in systemic lupus erythematosus (SLE), but limited studies have explored the role of LLDAS in lupus nephritis (LN). This study aims to investigate the frequency and predictors of LLDAS attainment and its benefit on LN relapse and renal function preservation in patients ...
Chak Kwan Cheung, Desmond Y. H. Yap, Ka Lai Lee, Philip H. Li, Iris Y. K. Tang, Chak Sing Lau, Shirley C. W. Chan   +6 more
wiley   +1 more source

Improvements in Health‐Related Quality of Life With Treat‐to‐Target Urate‐Lowering Therapy in Gout: A Post Hoc Analysis of a Randomized Multicenter Trial

Arthritis Care &Research, EarlyView.
Objective Although treat‐to‐target urate‐lowering therapy (ULT) is endorsed as best practice in gout management, limited data exist on its impact on health‐related quality of life (HRQoL). We assessed the impact of treat‐to‐target ULT on HRQoL among participants receiving protocolized gout care, identifying factors associated with HRQoL and HRQoL ...
Austin Barry, Bryant R. England, Harlan Sayles, Lindsay N. Helget, Maria Androsenko, Hongsheng Wu, Kaleb Michaud, Bridget Kramer, Jeff A. Newcomb, Mary T. Brophy, Anne Davis‐Karim, Ryan Ferguson, Michael H. Pillinger, Tuhina Neogi, Paul M. Palevsky, James R. O'Dell, Ted R. Mikuls   +16 more
wiley   +1 more source