Methods for Studying Treatments for Rare Diseases

Date: Tuesday, 09 24, 2019; Speaker: Joshua Gagne, Pharm.D., Sc.D., Associate Professor, Division of Pharmacoepidemiology and Pharmacoeconomics, Harvard T.H. Chan School of Public Health; Webinar;
Source: NIH Calendar of Events - Category: American Health Source Type: events

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The need to reform the clinical trials process with the patient in mind is becoming ever more apparent. Given the expense and time it takes to find and retain suitable trial candidates, making a trial scientifically rigorous is no longer enough. It must also be compatible with a patient ’s lifestyle.   Participants in clinical trials are not numbers on a spreadsheet. They are customers, consumers, patients and people with complexities, preferences and nuances. Increasingly they are demanding more choice and more involvement in their treatment journey.  New technology that enables pharma to digitise t...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news
CONCLUSION: This is the first study to examine such a large library of clinically approved compounds for the identification of novel drug candidates for MCL treatment, the results could be rapidly translated into clinical practice in patients with MCL. PMID: 31526347 [PubMed - as supplied by publisher]
Source: Combinatorial Chemistry and High Throughput Screening - Category: Chemistry Authors: Tags: Comb Chem High Throughput Screen Source Type: research
In this study, researchers studied 438,952 participants in the UK Biobank, who had a total of 24,980 major coronary events - defined as the first occurrence of non-fatal heart attack, ischaemic stroke, or death due to coronary heart disease. They used an approach called Mendelian randomisation, which uses naturally occurring genetic differences to randomly divide the participants into groups, mimicking the effects of running a clinical trial. People with genes associated with lower blood pressure, lower LDL cholesterol, and a combination of both were put into different groups, and compared against those without thes...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
ConclusionsThis first report of SBRT for pulmonary LCNEC demonstrates that SBRT is a feasible modality for this rare disease. A multidisciplinary thoracic oncology approach involving medical oncology, thoracic surgery, radiation oncology and pulmonology is strongly recommended to ensure proper patient selection for receipt of SBRT.
Source: Reports of Practical Oncology and Radiotherapy - Category: Cancer & Oncology Source Type: research
Multivitamins, drugs, gene therapies, human skin, heart, eyeballs, kidneys, entire dead bodies – everything comes with a price tag. Putting aside the moral questions of why and how come that the capitalist market priced even our body parts and health, we asked the question of how much is life worth: what is the maximum that you would/should pay for a life-saving drug? How high is too high a cost if a drug can save 200-300 babies a year from debilitating illness or death? And ultimately, does the pricing of new technologies, especially gene therapies, enable to fulfill their promise? There’s a price for ever...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Bioethics Biotechnology Future of Pharma Genomics cost daraprim drug drug price Gene gene therapy genetics insulin life medication pricing policy rare disease rare disorder Source Type: blogs
Abstract CD73 is a ubiquitously-expressed glycosylphosphatidylinositol (GPI)-anchored glycoprotein that converts extracellular adenosine 5'-monophosphate (AMP) to adenosine. Anti-CD73 inhibitory antibodies are currently undergoing clinical testing for cancer immunotherapy. However, CD73 has many protective physiological functions that need to be taken into account for new targeted therapies. This review covers CD73 functions in multiple organ systems and cell types, with a particular focus on novel findings from the last 5 years. Missense loss-of-function mutations in the CD73-encoding gene (NT5E) cause the rare d...
Source: Am J Physiol Cell Ph... - Category: Cytology Authors: Tags: Am J Physiol Cell Physiol Source Type: research
CONCLUSIONS: The diagnosis and treatment of peritoneal serous papillary carcinoma may be delayed or may not be performed unless Class III findings are detected through uterine mucosal cytology before surgery. Surgeons should not hesitate to perform laparotomy when necessary to identify and appropriately treat patients, even if abnormalities are not detected in the preoperative examination. PMID: 31448396 [PubMed - in process]
Source: Clinical Breast Cancer - Category: Cancer & Oncology Authors: Tags: Tokai J Exp Clin Med Source Type: research
This article examines the various types of perianal cancers and their precursor lesions and will explore treatment options as well as outcomes and prognosis. [...] Thieme Medical Publishers 333 Seventh Avenue, New York, NY 10001, USA.Article in Thieme eJournals: Table of contents  |  Abstract  |  Full text
Source: Clinics in Colon and Rectal Surgery - Category: Surgery Authors: Tags: Review Article Source Type: research
In this study, blood samples extracted by venipuncture from 30 healthy volunteers were used to obtain DNA from buffy coat and whole blood. The purity and integrity of the nucleic acids obtained were assessed by spectrophotometry, fluorimetry, and agarose electrophoresis, and functionality was assessed by PCR and real-time PCR. Another aspect tested in this study was based on the comparison between short-term and long-term storage at -80°C and fresh samples from both matrices to evaluate the storage conditions at the biobank. Results showed differences in the yield obtained from both matrices as a function of the storag...
Source: Biopreservation and Biobanking - Category: Biomedical Science Tags: Biopreserv Biobank Source Type: research
Changhua Wu, Liang Wang, Lei Guo, Lihong Zhang, Jing LiJournal of Cancer Research and Therapeutics 2019 15(4):947-952 Undifferentiated sarcoma (UNDS) of the soft tissue is an exceedingly rare disease. Its diagnosis depends mainly on molecular and immunohistochemical analyses to exclude other soft-tissue sarcomas. It is difficult to confirm a positive diagnosis by imaging pathological features because of their rarity and similarity with other conditions. Since 2013, only 13 cases of undifferentiated soft-tissue sarcoma, mostly diagnosed through imaging of solid tumors in infant and children, have been reported. The authors...
Source: Journal of Cancer Research and Therapeutics - Category: Cancer & Oncology Authors: Source Type: research
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