scholarly journals Rett Syndrome: Coming to Terms with Treatment

2014 ◽  
Vol 2014 ◽  
pp. 1-20 ◽  
Author(s):  
Alan Percy

Rett syndrome (RTT) has experienced remarkable progress over the past three decades since emerging as a disorder of worldwide proportions, particularly with discovery of the linkage of RTT to MECP2 mutations. The advances in clinical research and the increasing pace of basic science investigations have accelerated the pattern of discovery and understanding. Clinical trials are ongoing and others are planned. A review of these events and the prospects for continued success are highlighted below. The girls and women encountered today with RTT are, overall, in better general, neurologic, and behavioral health than those encountered earlier. This represents important progress worldwide from the concerted efforts of a broadly based and diverse clinical and basic research consortium as well as the efforts of parents, family, and friends.

1996 ◽  
Vol 8 (S1) ◽  
pp. 21-23 ◽  
Author(s):  
Jeffrey L. Cummings

Barriers to behavioral research are multifactorial. They include attitudinal, conceptual, financial, and administrative factors. In general, behavioral research and clinical research have received less support than basic science research (Marshall, 1994). The traditional emphasis has been on funding of basic research with the expectation that clinical research can be pursued without specific financial support. Although this might have been possible in the past, the increasingly tight control of clinicians' time and the need for cost-effective healthcare delivery have rendered this approach obsolete. Advances in clinically applicable information will require the type of research funding usually reserved for basic science endeavors.


PLoS ONE ◽  
2021 ◽  
Vol 16 (4) ◽  
pp. e0248898
Author(s):  
Joshua R. Wortzel ◽  
Brandon E. Turner ◽  
Brannon T. Weeks ◽  
Christopher Fragassi ◽  
Virginia Ramos ◽  
...  

Whereas time trends in the epidemiologic burden of US pediatric mental health disorders are well described, little is known about trends in how these disorders are studied through clinical research. We identified how funding source, disorders studied, treatments studied, and trial design changed over the past decade in US pediatric mental health clinical trials. We identified all US pediatric interventional mental health trials submitted to ClinicalTrials.gov between October 1, 2007 and April 30, 2018 (n = 1,019) and manually characterized disorders and treatments studied. We assessed trial growth and design characteristics by funding source, treatments, and disorders. US pediatric mental health trials grew over the past decade (compound annual growth rate [CAGR] 4.1%). The number of studies funded by industry and US government remained unchanged, whereas studies funded by other sources (e.g., academic medical centers) grew (CAGR 11.3%). Neurodevelopmental disorders comprised the largest proportion of disorders studied, and Non-DSM-5 (Diagnostic and Statistical Manual-5) conditions was the only disorder category to grow (14.5% to 24.6%; first half to second half of decade). There was significant growth of trials studying non-psycho/pharmacotherapy treatments (33.8% to 49.0%) and a decline in trials studying pharmacotherapies (31.7% to 20.6%), though these trends differed by funding source. There were also notable differences in funding sources and treatments studied within each disorder category. Trials using double blinding declined (26.2% to 18.0%). Limitations include that ClinicalTrials.gov is not an exhaustive list of US clinical trials, and trends identified may in part reflect changes in trial registration rather than changes in clinical research. Nevertheless, ClinicalTrials.gov is among the largest databases available for evaluating trends and patterns in pediatric mental health research that might otherwise remain unassessable. Understanding these trends can guide researchers and funding bodies when considering the trajectory of the field.


2010 ◽  
Vol 38 (2) ◽  
pp. 286-302 ◽  
Author(s):  
Patrick L. Taylor

Innovative therapy is the name we give to novel medical interventions, radically different from the standard of care, provided in order to benefit a patient, rather than to acquire new knowledge. They are paradigmshifting, not incremental, responses to serious patient problems that standard medical care inadequately addresses. Innovative therapies are often devised by clinicians, not basic science researchers; they do not follow the linear model of basic research, to translation, to clinical research, to application. Instead, they come from thinking backwards from the patient’s circumstances, and forward from deep knowledge of how the body functions, to challenge the limits of current mechanisms for effecting cures.


2020 ◽  
Author(s):  
Jun-hong Hu ◽  
Chen-Yu Wang ◽  
Shi-Can Zhou ◽  
Xing-Wang Li ◽  
Bing-Hui Li ◽  
...  

Abstract Background The aim of this bibliometric analysis was to evaluate the clinical trials of randomized controlled trials (RCTs) of colorectal cancer all the world over the past decade. Methods The PubMed and Web of Science database were searched to obtain a randomized controlled trial of colorectal cancer from January 1, 2008 to January 1, 2018.The included literatures were analyzed with the bibliometric analysis. Results In the past 10 years, Randomized controlled trials of colorectal cancer have shown an upward trend; Most of the top ten research institutions in the literature are from the USA, the UK and other regions which has the high incidence of colorectal cancer; Most of the related research journals are also sponsored by European and American countries; the frequently cited top 15 literatures are mostly international multi-center clinical research, and there are fewer participants in Chinese research institutions. By keyword co-occurrence, colorectal cancer research mostly involves screening, disease-free survival, drug treatment, surgical methods, clinical trials, quality of life and prognosis; The results of the co-authorship network analysis show that Chinese researchers are less involved in international cooperation. Conclusions High-quality randomized controlled trials are increasingly favored by top international journals. However, there is still a large gap in clinical research between China and abroad. Researchers should gradually implement the standardization and accuracy of clinical trials, strengthen international multi-center cooperation and emphasize quality control.


Author(s):  
Murugan Panchatcharam ◽  
Sravanthi Lakshmi Mukku

Clinical research involves working with human subjects to answer questions relevant to their well-being in an ethical manner. The current scenario from the past one year has drastically changed the face of the clinical trials. The present COVID prevalence and simultaneously conducting the research with all the regulations and the precautions has been the difficult task for the contract research organisations (CRO).


2021 ◽  
Vol 11 ◽  
Author(s):  
Zeyu Zhang ◽  
Zhiming Wang ◽  
Yun Huang

IntroductionCholangiocarcinoma (CCA) is the second most common hepatic malignancy. Progress and developments have also been made in the field of CCA management along with increasing scientific publications during the past decades, which reflect topics of general interest and suggest the future direction of studies. The purpose of this bibliometric study is to summarize scientific publications during the past 25 years in the field of CCA using a machine learning method.Material and MethodsScientific publications focusing on CCA from 1995 to 2019 were searched in PubMed using the MeSH term “cholangiocarcinoma.” Full associated data were downloaded in the format of PubMed and extracted in the R platform. Latent Dirichlet allocation (LDA) was adopted to identify the research topics from the abstract of each publication using Python.ResultsA total of 8,276 publications related to CCA from the last 25 years were found and included in this study. The most type of publications remained little changed, while the proportion of clinical trials remained relatively low (7.24% as the highest) and, more significantly, with a further downward trend during the recent years (1.42% in 2019). Neoplasm staging, hepatectomy, and survival rate were the most concerning terms among those who are diagnosis-related, treatment-related, and prognosis-related. The LDA analyses showed chemotherapy, hepatectomy, and stent as the highly concerned research topics of CCA treatment. Meanwhile, conversions from basic studies to clinical therapies were suggested by a poor connection between clusters of treatment management and basic research.ConclusionThe number of publications of CCA has increased rapidly during the past 25 years. Survival analysis, differential diagnosis, and microRNA expression are the most concerned topics in CCA studies. Besides, there is an urgent need for high-quality clinical trials and conversions from basic studies to clinical therapies.


Author(s):  
Gianfranco Pacchioni

This chapter illustrates with examples how science and technology have changed our way of life. We live longer, we live better, although this is not always appreciated. A brief description of life in Edwardian London of the early 1900s provides an idea of the changes that have occurred over the past century. Reasons why basic research is useful are illustrated to give a positive feeling about the role of science. The time required to transform basic research into valuable applications, however, can be very long. How a fundamental discovery in basic science has turned into magnetic resonance diagnostics in medicine is provided as an example.


2009 ◽  
Vol 24 (S1) ◽  
pp. 1-1
Author(s):  
F. Versonnen

Basic and clinical research are both of importance for psychiatry. In psychiatric care, both neurobiological and psychosocial underpinnings of clinical diagnosis and treatment. To come to a meaningful intervention, one is in need of different levels of explanation. Moreover, in a clinical setting, an integration of the so called Evidence Based Medicine (EBM) and Narrative Based Medicine (NBM) seems to be most appropriate. Basic research is sometimes seen as ‘more scientific’ and this may be reflected by granting more awards for these posters. A simple quantitative analysis is done by comparing the basic and clinical posters at the 20th ECNP Congress in Vienna. The total of submissions, the basic versus clinical ‘awards"- and Odds-ratio are calculated. Some corrections are needed. First, 10 psychology posters, were classified as basic and were reattributed to the clinical category. Second, after a qualitative analysis and correction for preclinical narratives or basic research methods for 7 awarded clinical posters, they were reclassified to the basic science category. All awarded basic posters were labeled correctly. After this double correction the Odds ratio was calculated. A total of 725 posters were submitted, 426 clinical and 299 basic. Fifty posters were awarded. The Odds ratio of getting an award was 5, 8 in favor of basic science posters. This is an expression of the current undervaluation of clinical research.


2020 ◽  
Vol 14 (4) ◽  
Author(s):  
Jerzy B. Gajewski ◽  
Magdy M. Hassouna ◽  
Jacques Corcos ◽  
Gary J. Gray ◽  
Le Mai Tu ◽  
...  

Sacral neuromodulation (SNM) has been used for the past 30 years, with significant improvements in the implantation technique and technological advances over the last several years. Canadian centers were involved with this technique from the beginning by participating in several multicenter clinical trials and engaging in basic and clinical research. Presently, six Canadian centers continue to have SNM implantation programs.


Author(s):  
Abdelrahman H. Abdelmoneim ◽  
Safinaz I. Khalil ◽  
Hiba Awadelkareem Osman Fadl ◽  
Ayesan Rewane ◽  
Sahar G. Elbager

Background: COVID-19 pandemic has dramatically engulfed the world causing catastrophic damage to human society. Several therapeutic and vaccines have been suggested for the disease in the past months, with over 150 clinical trials currently running or under process. Nevertheless, these trials are extremely expensive and require a long time, which presents the need for alternative cost-effective methods to tackle this urgent requirement for validated therapeutics and vaccines. Bearing this in mind, here we assess the use of in silico clinical trials as a significant development in the field of clinical research, which holds the possibility to reduce the time and cost needed for clinical trials on COVID-19 and other diseases. Methods: Using the PubMed database, we analyzed six relevant scientific articles regarding the possible application of in silico clinical trials in testing the therapeutic and investigational methods of managing different diseases. Results: Successful use of in silico trials was observed in many of the reviewed evidence. Conclusion: In silico clinical trials can be used in refining clinical trials for COVID-19 infection. Keywords: in silico, clinical trials, COVID-19, SARS-CoV-2, vaccine How


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