screening techniques
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2021 ◽  
pp. 59-70
Author(s):  
Marcia Scheiner ◽  
Joan Bogden
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2021 ◽  
Author(s):  
Moataz Dowaidar

High throughput screening techniques have facilitated the creation of RNA treatments, and these findings have been accompanied by unique modifications of RNA structures to increase stability and minimize toxicity, as well as innovative delivery systems such as LNPs. "undruggable" targets, "swiftly construct vaccinations," and to provide therapeutic approaches that can be tailored to individual patients Like the genetic code, RNA therapies hold great promise.At the same time, our hospital launched a specialized RNA Therapeutics program that assists both our internal scientists and external academic groups or small firms aiming to apply their fundamental research discoveries to innovative treatments. mRNA is primarily stable because of our primary study in sequencing and structure. For synthesis, purification, and validation of RNA constructs, we have a contemporary GMP program with clean rooms. Our company collaborates with Nanomedicine to build LNP structures that transport mRNA constructs locally and systemically. We have an opportunity to hire and integrate first-in-man clinical trials unit, as well as a big hospital system with a clinical research infrastructure that enables industry-sponsored Phase 2 and 3 clinical trials. Additionally, we have a large-scale mRNA construction industrial sponsor for Phases 2, 3, and commercialization. Essentially, we've put up a production line for universities and small firms interested in turning their mRNA creations into revolutionary medications. One hospital, one point of entry to ensure that RNA-based drugs are created and translated into the clinic efficiently. Many tiny firms with fresh concepts for disruptive mRNA therapeutics cannot reach the clinic without new translational capabilities. We think we are the only academic organization with the infrastructure to help small academic groups and biotech enterprises, and we want to aid both internal and external translational activities in the coming years.


Author(s):  
Firooz Esmaeilzadeh ◽  
Batoul Ahmadi ◽  
Sajad Vahedi ◽  
Saeed Barzegari ◽  
Abdolhalim Rajabi

Background: Beta-thalassemia minor and thalassemia major are an autosomal recessive disease with hypochromic, microcytic anemia, and morbidities, Today, therapeutic advances have significantly improved the life expectancy of thalassemia major patients, but at the cost of financial toxicity. The present study aimed to investigate the possibility of increasing the funding for thalassemia screening programs and comparing the cost-effectiveness of screening for thalassemia in the treatment of the patients. Methods: In this study, screening for thalassemia minor was compared with the treatment of thalassemia major patients. A decision tree model was used for analysis. A hospital database, supplemented with a review of published literature, was used to derive input parameters for the model. A lifetime study horizon was used and future costs and consequences were discounted at 3%. The approach of purchases of services was used to evaluate the screening test costs for patients with thalassemia major. Also, a bottom-up method was applied to estimate other screening and treatment costs. All the costs were calculated over one year. The number of gained quality-adjusted life years (QALYs) was calculated using the EQ-5D questionnaire in the evaluated patients. Results: In this study, 26.97 births of patients with thalassemia major were prevented by screening techniques. On the other hand, total screening costs for patients with thalassemia major were estimated equal to US$ 879879, while the costs of preventing the birth of each thalassaemia major patient was US$ 32 624 by screening techniques. In comparison, the cost of managing a patient with thalassemia major is about US$ 136 532 per year. The life time QALYs for this is 11.8 QALYs. Results are presented using a societal perspective. Incremental cost per QALY gained with screening as compared with managing thalassaemia major was US$ 11 571. Conclusion: Screening is a long-term value for money intervention that is highly cost effective and its long-term clinical and economic benefits outweigh those of managing thalassaemia major patients.


2021 ◽  
pp. 6-16
Author(s):  
Judith  Dekker ◽  
Isabelle Hooijer ◽  
Johannes C.F. Ket ◽  
Aleksandra Vejnović ◽  
Giuseppe Benagiano ◽  
...  

<b><i>Objective:</i></b> Based on the hypothesis that neonatal uterine bleedings (NUB), occurring mostly in the first week after birth, could represent a pathogenetic mechanism for early-onset endometriosis, this systematic review (SR) was undertaken to evaluate the prevalence and screening strategies used to assess and quantify NUB. <b><i>Design:</i></b> Both a SR and a sample literature search in PubMed and Embase were conducted to gather information on NUB prevalence and screening techniques. This was performed by an information specialist. Only full-text articles regarding the assessment of NUB in neonates in the first 2 weeks after birth were included. No limit on language or publication data was used. <b><i>Materials and Methods:</i></b> The SR was registered in PROSPERO (CRD42019138121). Data was first assessed for eligibility on title and abstract by 2 blinded review authors. Any disagreements were discussed with a third reviewer if necessary. Subsequently, full-text articles were read and assessed for quality using the Cochrane Collaboration Handbook. <b><i>Results:</i></b> Out of 1,988 articles in the systematic search, 10 relevant articles were selected, of which 8 were identified through the systematic search and 2 were found through other sources. The sample search of 4,445 articles did not bring up relevant articles. Results were not comparable due to the heterogeneity of screening techniques, although data showed consensus. The prevalence of visible bleeding ranged from 3.3 to 53.8% and the prevalence of occult bleeding from 25.4 to 96.7%. The occurrence was the highest between the 3rd and 7th day postpartum (PP) and the bleeding lasted for 3–4 days on average. Various screening techniques for detecting NUB were found in the literature, including the use of hemoglobin detection devices (such as Hemastix) in the vaginal vestibulum, comparison of diapers with stains of known volume, colposcopy, and ultrasonography. <b><i>Conclusion:</i></b> The reported prevalence of NUB varies considerably, with a consistent occurrence between the 3rd and the 7th day PP. Literature to assess NUB is dated. The techniques are poorly described and heterogeneous. Future research should focus on prospective cohort studies in order to attempt to correlate NUB cases to (early-onset) endometriosis.


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