New Developments and Challenges in Rare Genitourinary Tumors: Non-Urothelial Bladder Cancers and Squamous Cell Cancers of the Penis

2017 ◽  
pp. 330-336
Author(s):  
Jeanny B. Aragon-Ching ◽  
Lance C. Pagliaro
Keyword(s):  
Clinical Trials ◽  
Squamous Cell ◽  
Rare Diseases ◽  
Research Funding ◽  
Diagnosis And Treatment ◽  
New Developments ◽  
Effective Care ◽  
Recent Developments ◽  
Urothelial Bladder ◽  
Genitourinary Cancers

The diagnosis and treatment of rare genitourinary tumors is inherently challenging. The Rare Diseases Act of 2002 initially defined a rare disorder as one that affects fewer than 200,000 Americans. The lack of widely available clinical guidelines, limited research funding, and inaccessible clinical trials often lead to difficulty with treatment decisions to guide practitioners in rendering effective care for patients with rare genitourinary cancers. This article will discuss basic tenets of diagnosis and treatment as well as recent developments and clinical trials in rare non-urothelial bladder cancers and penile squamous cell cancers.

Cystic Fibrosis: Recent Developments in Diagnosis and Treatment

1986 ◽  
Vol 7 (9) ◽  
pp. 276-286
Author(s):  
Robert C. Stern
Keyword(s):  
Cystic Fibrosis ◽  
Life Expectancy ◽  
Genetic Defect ◽  
Diagnosis And Treatment ◽  
New Developments ◽  
Recent Developments ◽  
Extended Life

Advances in diagnosis and treatment of cystic fibrosis have greatly extended life expectancy and have decreased morbidity. However, further progress requires knowledge of the fundamental genetic defect and how it is related to the pulmonary pathophysiology. Major new developments in both these areas are likely within the next 5 to 10 years.

scholarly journals Participant-funded clinical trials on rare diseases

2020 ◽  
Vol 93 (4) ◽  
pp. 267.e1-267.e9
Author(s):  
Rafael Dal-Ré ◽  
Francesc Palau ◽  
Encarna Guillén-Navarro ◽  
Carmen Ayuso
Keyword(s):  
Clinical Trials ◽  
Rare Diseases

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

Collaborative Research Funding

2014 ◽  
Vol 36 (4) ◽  
Author(s):  
Markus Behnke ◽  
Laura McConnell ◽  
Chris Ober
Keyword(s):  
International Collaboration ◽  
Research Funding ◽  
International Committee ◽  
Research Projects ◽  
Scientific Success ◽  
Funding Sources ◽  
Chemistry Research ◽  
National Organizations ◽  
Recent Developments ◽  
History Of

Within a changing research world, international collaboration has become even more important in achieving scientific success. Given the increased need and desire for multinational research, the actors are forced to identify appropriate funding sources. Whereas, science knows no international boundaries, support for scientific research, including in chemical sciences, is mostly provided by the national funding organizations. This is particularly true for the chemical sciences, where most research projects are relatively small in size and with respect to the number of involved PIs. Traditionally, national organizations are reluctant to provide funds to non-domestic researchers, and in practice, funding truly international research projects can be a real challenge for a variety of technical and bureaucratic reasons. In an effort to change this, an international Committee on Chemistry Research Funding (CCRF)—backed by several leading funding organizations—was established by IUPAC in December 2007 to promote increased international collaboration and networking in the global chemistry community. The following report gives a short overview on the history of IUPAC’s involvement in service for chemistry research funding and on the most recent developments.

scholarly journals What We Learn from the CTAD 2018 (Clinical Trials Alzheimer’s Disease)

2018 ◽  
pp. 1-2
Author(s):  
B. Vellas ◽  
P. Aisen ◽  
M. Weiner ◽  
J. Touchon
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Safety Data ◽  
Phase Iii ◽  
Drug Trials ◽  
New Developments ◽  
Fda Guidance ◽  
Clinical Biomarkers ◽  
Early Alzheimer’S Disease

We are happy to publish the CTAD 2018 abstracts in the present JPAD issue. As you can see many new interesting studies are presented in this issue of the journal: from new drug trials to biomarkers, imaging studies, as well as new clinical outcomes. More specifically, we will have several hot topics presentation on: 1. Major drug trials using bace inhibitors (verubecestat, lanabecestat, atabecestat, elenbecestat…) in the early phase of the disease (APECS early trials…). Both clinical, biomarkers (MRI, CSF, PET) and safety data will be presented. 2. New data on blood biomarkers including a keynote from R. Bateman, and presentations from Araclon and Roche biomarkers. 3. Results from phase III and IIB trials including a novel and multi-targeted oligosaccharide in patients with mild-moderate AD in China; the AMBAR (Alzheimer’s Management By Albumin Replacement) study, the TOMMORROW trial: a trial to delay the onset of MCI due to AD and qualify a genetic biomarker algorithm, the 18-month STEADFAST trial of azeliragon in participants with mild Alzheimer’s Disease; a longitudinal 148-week extension 4. Results 18 from F-AV-1451-A16: a clinicopathological study of the correspondence between flortaucipir PET imaging and post-mortem assessment of tau pathology. 5. Latest developments in anti-amyloid monoclonal antibodies including aducanumab nonnegligible, and new results and data analyses of the BAN2401 study 201 in early AD. 6. New developments with safety and efficacy of lemborexant for sleep-wake regulation in patients with irregular sleep-wake rhythm disorders and Alzheimer’s Disease dementia. 7. Advances with the ABBV-8E12, a humanized anti-tau monoclonal antibody, for the treatment of early Alzheimer’s Disease. 8. Endpoints for early Alzheimer’s Disease clinical trials: interpretation and application of the draft FDA guidance. And many others… It is important to underline that a not negligible number of abstracts concern non amyloid targets (eg: Tau-related targets but also targets outside the classical AD cascade).

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