Results 311 to 320 of about 5,566,486 (385)

Long‐Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objectives This ongoing, open‐label extension study is evaluating the long‐term safety, tolerability, and efficacy of givinostat, a Class I and II histone deacetylase inhibitor, in patients with Duchenne muscular dystrophy (DMD). Methods The recruited patients completed one of two prior clinical studies (one Phase 2 and one Phase 3 [EPIDYS ...
Craig M. McDonald, Michela Guglieri, Dragana Vučinić, Gyula Acsadi, John F. Brandsema, Claudio Bruno, Erika L. Finanger, Amy Harper, Mercedes Lopez Lobato, Riccardo Masson, Nuria Muelas, Francina Munell, Yoram Nevo, Yann Péréon, Han Phan, Valeria A. Sansone, Mariacristina Scoto, Tracey Willis, Richard S. Finkel, Krista Vandenborne, Sara Cazzaniga, Silvia Montrasio, Federica Alessi, Paolo Bettica, Eugenio Mercuri, for the Givinostat Study 51 Investigators, the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) Investigators, the ImagingDMD Investigators, Enrico Bertini, Giacomo Pietro Comi, Eugenio Maria Mercuri, Giuseppe Vita, Sonia Messina, Claudio Bruno, Riccardo Masson, Valeria Sansone, Nathalie Goemans, Liesbeth De Waele, Laurent Servais, Teresa Gidaro, Odile Boespflug‐Tanguy, Yann Péréon, Jessika Johannsen, Astrid Blaschek, Ulrike Schara‐Schmidt, Erik Niks, Imelda de Groot, Saskia Houwen‐van Opstal, Andres Nascimento, Juan Jesus Vilchez, Nuria Muelas, Francina Munell, Marcos Madruga Garrido, Mercedes Lopez Lobato, Michaela Guglieri, Tracey Willis, Stefan Spinty, Daniel Hawcutt, Mariacristina Scoto, Jean K. Mah, Laura McAdam, Kathryn Selby, Katherine Mathews, Craig McDonald, Craig Zaidman, Barry Byrne, John Brandsema, Gyula Acsadi, Chamindra Laverty, Amy Harper, Erika Finanger, Han Phan, Yoram Nevo, Vedrana Milic Rasic, Dragana Vucinic   +74 more
wiley   +1 more source

SYHA1813, A VEGFR and CSF1R Inhibitor, in Patients With Recurrent High‐Grade Gliomas: A Multicenter, Open‐Label Phase I Study

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Recurrent high‐grade gliomas have a poor prognosis and limited therapeutic options. This study aimed to evaluate the safety and efficacy of SYHA1813, a dual inhibitor of VEGFR and CSF1R, in patients with recurrent high‐grade gliomas.
Zhuang Kang, Shenglan Li, Liang Wang, Qianxue Chen, Weiguo Hu, Ting Lei, Ying Mao, Jing Zhang, Xiaojun Xiang, Qiming Wang, Zhengwen He, Tao Sun, Yulei Wang, Mengqian Huang, Rong Zhang, Feng Chen, Wenbin Li   +16 more
wiley   +1 more source

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Use of Single-Arm Trials for US Food and Drug Administration Drug Approval in Oncology, 2002-2021.

JAMA Oncology, 2022
Importance Single-arm trials have allowed for transformative therapies to be made available to patients expeditiously. However, using single-arm trials to support drug approval presents several challenges that must be carefully considered ...
S. Agrawal, Shaily Arora, L. Amiri-Kordestani, R. A. de Claro, L. Fashoyin-Aje, N. Gormley, Tamy Kim, S. Lemery, Gautam U. Mehta, E. Scott, H. Singh, Shenghui Tang, M. Theoret, R. Pazdur, P. Kluetz, J. Beaver   +15 more
semanticscholar   +1 more source

Impact of Drug Approval Pathways for Paediatric Inflammatory Bowel Disease.

Journal of Crohn's & Colitis, 2021
BACKGROUND AND AIMS Timely access to approved medications is a priority in paediatric inflammatory bowel disease (IBD). To date, the timing of drug studies in paediatric IBD has been suboptimal with most studies conducted long after approval has been ...
E. Crowley, Christopher Ma, Marija Andic, B. Feagan, A. Griffiths, V. Jairath   +5 more
semanticscholar   +1 more source

Overall Survival, Progression-Free Survival, and Tumor Response Benefit Supporting Initial US Food and Drug Administration Approval and Indication Extension of New Cancer Drugs, 2003-2021

Journal of Clinical Oncology, 2022
PURPOSE Clinical trial evidence is routinely evaluated for initial drug approvals, yet the benefit of indication extensions remains uncertain. This study evaluates the clinical benefit supporting new cancer drugs' initial and supplemental US Food and ...
D. Michaeli, T. Michaeli
semanticscholar   +1 more source

Drug Approval and Drug Effectiveness

Annual Review of Clinical Psychology, 2014
Data on the efficacy and safety of psychiatric medicines should form the foundation of evidence-based treatment practices. The US Food and Drug Administration (FDA) reviews such data in determining whether to approve new treatments, and the published literature serves as a repository for evidence on treatment benefits and harms.
Glen I. Spielmans, Irving Kirsch
openaire   +3 more sources

Drug Approval Highlights for 2003

The Nurse Practitioner, 2004
In the past 12 months, the FDA has approved important new pharmaceutical drugs and devices of particular interest to primary health care providers. The drugs include: Oxytrol (for urinary incontinence), Valtrex (for reducing the risk of heterosexual transmission of genital herpes), Femring (for vaginal delivery of hormone therapy), Uroxatral (for ...
Laustsen, Gary, Wimett, Lynn
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Polypharmacology of Approved Anticancer Drugs

Current Drug Targets, 2017
The major drug discovery efforts in oncology have been concentrated on the development of selective molecules that are supposed to act specifically on one anticancer mechanism by modulating a single or several closely related drug targets. However, a bird's eye view on data from multiple available bioassays implies that most approved anticancer agents ...
Amelio, Ivano, Lisitsa, Andrey, Knight, Richard A., Melino, Gennaro, Antonov, Alexey V.   +4 more
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Approving new drugs

Science, 2017
As the Trump administration takes shape, there is much speculation as to what major changes will be made. A dominant theme of the Trump campaign was to cut through bureaucracy in Washington, D.C., thereby enhancing innovation and bringing new technology to Americans more quickly.
openaire   +3 more sources