| 1. |
|
|
| 2. |
- Mercuri, E., et al.
(author)
-
Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study
- 2020
-
In: Journal of Comparative Effectiveness Research. - : Becaris Publishing Limited. - 2042-6305 .- 2042-6313. ; 9:5, s. 341-360
-
Journal article (peer-reviewed)abstract
- Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren benefit correlations and ataluren safety. Patients & methods: Propensity score matching was performed to identify STRIDE and CINRG DNHS patients who were comparable in established disease progression predictors (registry cut-off date, 9 July 2018). Results & conclusion: Kaplan-Meier analyses demonstrated that ataluren + SoC significantly delayed age at loss of ambulation and age at worsening performance in timed function tests versus SoC alone (p <= 0.05). There were no DMD genotype-phenotype/ataluren benefit correlations. Ataluren was well tolerated. These results indicate that ataluren + SoC delays functional milestones of DMD progression in patients with nmDMD in routine clinical practice. ClinicalTrials.gov identifier: NCT02369731. ClinicalTrials.gov identifier: NCT02369731.
|
|
| 3. |
- Arnetz, Bengt B., et al.
(author)
-
War-related mental health disorders among Iraqis 10 years after the 1991 Gulf War : A comparative study of soldiers and civilians living under sustained socio-environmental stress
- 2009
-
In: The New Iraqi Journal of Medicine. - 1817-5562. ; 5:1, s. 9-21
-
Journal article (peer-reviewed)abstract
- Background: Prior studies of mental health consequences of the Gulf War (GW) have been confined to Allied forces, limiting the ability to control for important geographically and culturally-related factors. We conducted an epidemiological mental health study among Iraqi soldiers and civilians who are still residing in Iraq. This group has been exposed to sustained socio-environmental stress. Methods: A cross-sectional sample of 742 Iraqi GW veterans and 413 civilians responded to a validated mental health survey. The response rate was 96.3%. Mental health disorders, including post traumatic stress disorder (PTSD), were classified using both self-reports and validated scales. War-related exposure was calculated using the sum score of items assessing trauma exposure. Results: Iraqi soldiers reported significantly more depression (Odds Ratio [OR] 4.9; 95% confidence interval [CI] 2.2-11.1) and anxiety (OR 3.9; 95% CI, 1.2-13.3) compared to civilians, adjusting for age, education, and smoking. Soldiers closest to Kuwait during the GW reported significantly more depression compared to soldiers deployed further away from the war epicenter (OR 104.6; 95% CI, 28.0-390.8) and anxiety (OR 4.1; 95% CI, 1.5-11.1). The highest self-reported trauma exposure occurred in the southwest of Iraq. Conclusion: Iraqi soldiers that took part in the GW are at increased risk suffering from many of the same mental health disorders plaguing Allied soldiers. Soldiers closest to Kuwait were more at risk, suggesting a direct link to war-specific environmental exposures, although self-reported trauma exposure was higher in the southwest of Iraq. The study offers additional insights into the mental health consequences of living under sustained socio-environmental stress, originating from the Iraqi war. The study points out socio-environmental factors worthy of further explorations.
|
|
| 4. |
- Grzybowski, Andrzej, et al.
(author)
-
Endophthalmitis Prophylaxis in Cataract Surgery : Overview of Current Practice Patterns Around the World
- 2017
-
In: Current pharmaceutical design. - : BENTHAM SCIENCE PUBL LTD. - 1381-6128 .- 1873-4286. ; 23:4, s. 565-573
-
Research review (peer-reviewed)abstract
- Background: Acute-onset postoperative endophthalmitis after cataract surgery remains a rare but important cause of visual loss. There is no global consensus regarding the optimal strategies for prophylaxis of endophthalmitis and practices vary substantially around the world, especially with respect to the use of intracameral antibiotics. The European Society of Cataract & Refractive Surgeons in a randomized clinical trial (2007) reported an approximately 5-fold reduction in endophthalmitis rates associated with the use of intracameral cefuroxime. Despite this report, the use of intracameral antibiotics has not been universally adopted. Methods: Various endophthalmitis prophylaxis patterns around the world (including the United States, Canada, Australia/New Zealand, Japan, China, India, Indonesia, South Africa, Argentina, Russia, Sweden and Mexico) are compared. Each contributing author was asked to provide similar information, including endophthalmitis rates based on published studies, current practice patterns, and in some cases original survey data. Various methods were used to obtain this information, including literature reviews, expert commentary, and some new survey data not previously published. Results: Many different practice patterns were reported from around the world, specifically with respect to the use of intracameral antibiotics. Conclusion: There is no worldwide consensus regarding endophthalmitis prophylaxis with cataract surgery.
|
|
| 5. |
- Sun, Jian-Ke, et al.
(author)
-
Enhancing crystal growth using polyelectrolyte solutions and shear flow
- 2020
-
In: Nature. - : Springer Science and Business Media LLC. - 0028-0836 .- 1476-4687. ; 579:7797, s. 73-79
-
Journal article (peer-reviewed)abstract
- The ability to grow properly sized and good quality crystals is one of the cornerstones of single-crystal diffraction, is advantageous in many industrial-scale chemical processes(1-3), and is important for obtaining institutional approvals of new drugs for which high-quality crystallographic data are required(4-7). Typically, single crystals suitable for such processes and analyses are grown for hours to days during which any mechanical disturbances-believed to be detrimental to the process-are carefully avoided. In particular, stirring and shear flows are known to cause secondary nucleation, which decreases the final size of the crystals (though shear can also increase their quantity(8-14)). Here we demonstrate that in the presence of polymers (preferably, polyionic liquids), crystals of various types grow in common solvents, at constant temperature, much bigger and much faster when stirred, rather than kept still. This conclusion is based on the study of approximately 20 diverse organic molecules, inorganic salts, metal-organic complexes, and even some proteins. On typical timescales of a few to tens of minutes, these molecules grow into regularly faceted crystals that are always larger (with longest linear dimension about 16 times larger) than those obtained in control experiments of the same duration but without stirring or without polymers. We attribute this enhancement to two synergistic effects. First, under shear, the polymers and their aggregates disentangle, compete for solvent molecules and thus effectively 'salt out' (that is, induce precipitation by decreasing solubility of) the crystallizing species. Second, the local shear rate is dependent on particle size, ultimately promoting the growth of larger crystals (but not via surface-energy effects as in classical Ostwald ripening). This closed-system, constant-temperature crystallization driven by shear could be a valuable addition to the repertoire of crystal growth techniques, enabling accelerated growth of crystals required by the materials and pharmaceutical industries.
|
|
