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https://www.readbyqxmd.com/read/28728956/ataluren-in-patients-with-nonsense-mutation-duchenne-muscular-dystrophy-act-dmd-a-multicentre-randomised-double-blind-placebo-controlled-phase-3-trial
#1
Craig M McDonald, Craig Campbell, Ricardo Erazo Torricelli, Richard S Finkel, Kevin M Flanigan, Nathalie Goemans, Peter Heydemann, Anna Kaminska, Janbernd Kirschner, Francesco Muntoni, Andrés Nascimento Osorio, Ulrike Schara, Thomas Sejersen, Perry B Shieh, H Lee Sweeney, Haluk Topaloglu, Már Tulinius, Juan J Vilchez, Thomas Voit, Brenda Wong, Gary Elfring, Hans Kroger, Xiaohui Luo, Joseph McIntosh, Tuyen Ong, Peter Riebling, Marcio Souza, Robert J Spiegel, Stuart W Peltz, Eugenio Mercuri
BACKGROUND: Duchenne muscular dystrophy (DMD) is a severe, progressive, and rare neuromuscular, X-linked recessive disease. Dystrophin deficiency is the underlying cause of disease; therefore, mutation-specific therapies aimed at restoring dystrophin protein production are being explored. We aimed to assess the efficacy and safety of ataluren in ambulatory boys with nonsense mutation DMD. METHODS: We did this multicentre, randomised, double-blind, placebo-controlled, phase 3 trial at 54 sites in 18 countries located in North America, Europe, the Asia-Pacific region, and Latin America...
July 17, 2017: Lancet
https://www.readbyqxmd.com/read/28727481/leucocyte-and-platelet-rich-fibrin-l-prf-as-a-regenerative-medicine-strategy-for-the-treatment-of-refractory-leg-ulcers-a-prospective-cohort-study
#2
Nelson R Pinto, Matias Ubilla, Yelka Zamora, Verónica Del Rio, David M Dohan Ehrenfest, Marc Quirynen
Chronic wounds (VLU: venous leg ulcer, DFU: diabetic foot ulcer, PU: pressure ulcer, or complex wounds) affect a significant proportion of the population. Despite appropriate standard wound care, such ulcers unfortunately may remain open for months or even years. The use of leukocyte- and platelet-rich fibrin (L-PRF) to cure skin ulcers is a simple and inexpensive method, widely used in some countries but unknown or neglected in most others. This auto-controlled prospective cohort study explored and quantified accurately for the first time the adjunctive benefits of topical applications of L-PRF in the management of such refractory ulcers in a diverse group of patients...
July 20, 2017: Platelets
https://www.readbyqxmd.com/read/28725432/transplanters-drive-cars-to-the-clinic-by-brewing-ice-t-the-moffitt-roadmap
#3
EDITORIAL
Frederick L Locke, Claudio Anasetti
Recent single institution clinical trial successes with anti-CD19 Chimeric Antigen Receptor (CAR) T cell therapy for B cell malignancies attracted significant attention from industry. Our center sought to rapidly and safely bring industry sponsored pivotal trials to our patients and to prepare for additional cell therapy trials in solid and liquid tumors from both industry and our own investigators. We implemented a collaborative cross departmental program to provide clinical care and trial coordination for immune cell therapies...
2017: Journal for Immunotherapy of Cancer
https://www.readbyqxmd.com/read/28716155/transplanters-drive-cars-to-the-clinic-by-brewing-ice-t-the-moffitt-roadmap
#4
EDITORIAL
Frederick L Locke, Claudio Anasetti
Recent single institution clinical trial successes with anti-CD19 Chimeric Antigen Receptor (CAR) T cell therapy for B cell malignancies attracted significant attention from industry. Our center sought to rapidly and safely bring industry sponsored pivotal trials to our patients and to prepare for additional cell therapy trials in solid and liquid tumors from both industry and our own investigators. We implemented a collaborative cross departmental program to provide clinical care and trial coordination for immune cell therapies...
July 18, 2017: Journal for Immunotherapy of Cancer
https://www.readbyqxmd.com/read/28705252/efficacy-of-prospective-pharmacogenetic-testing-in-the-treatment-of-major-depressive-disorder-results-of-a-randomized-double-blind-clinical-trial
#5
Víctor Pérez, Ariana Salavert, Jordi Espadaler, Miquel Tuson, Jerónimo Saiz-Ruiz, Cristina Sáez-Navarro, Julio Bobes, Enrique Baca-García, Eduard Vieta, José M Olivares, Roberto Rodriguez-Jimenez, José M Villagrán, Josep Gascón, Josep Cañete-Crespillo, Montse Solé, Pilar A Saiz, Ángela Ibáñez, Javier de Diego-Adeliño, José M Menchón
BACKGROUND: A 12-week, double-blind, parallel, multi-center randomized controlled trial in 316 adult patients with major depressive disorder (MDD) was conducted to evaluate the effectiveness of pharmacogenetic (PGx) testing for drug therapy guidance. METHODS: Patients with a CGI-S ≥ 4 and requiring antidepressant medication de novo or changes in their medication regime were recruited at 18 Spanish public hospitals, genotyped with a commercial PGx panel (Neuropharmagen®), and randomized to PGx-guided treatment (n = 155) or treatment as usual (TAU, control group, n = 161), using a computer-generated random list that locked or unlocked psychiatrist access to the results of the PGx panel depending on group allocation...
July 14, 2017: BMC Psychiatry
https://www.readbyqxmd.com/read/28700211/polyphenolic-polymersomes-of-temperature-sensitive-poly-n-vinylcaprolactam-block-poly-n-vinylpyrrolidone-for-anticancer-therapy
#6
Veronika Kozlovskaya, Fei Liu, Bing Xue, Fahim Ahmad, Aaron Alford, Mohammad Saeed, Eugenia Kharlampieva
We report a versatile synthesis for polyphenolic polymersomes of controlled submicron (< 500 nm) size for intracellular delivery of high and low molecular weight compounds. The nanoparticles are synthesized by stabilizing the vesicular morphology of thermally responsive poly(N-vinylcaprolactam)n-poly(N-vinylpyrrolidone)m (PVCLn-PVPONm) diblock copolymers with tannic acid (TA), a hydrolyzable polyphenol, via hydrogen bonding at the temperature above the copolymer's LCST. The PVCL179-PVPONm diblock copolymers are produced by controlled RAFT polymerization of PVPON using PVCL as a macro-chain transfer agent...
July 12, 2017: Biomacromolecules
https://www.readbyqxmd.com/read/28696079/impact-of-protease-inhibitor-based-anti-retroviral-therapy-on-outcomes-for-hiv-kidney-transplant-recipients
#7
Deirdre Sawinski, Brittany A Shelton, Shikha Mehta, Rhiannon D Reed, Paul A MacLennan, Sally Gustafson, Dorry L Segev, Jayme E Locke
Excellent outcomes have been demonstrated among select HIV-positive kidney transplant (KT) recipients with well-controlled infection, but to date no national study has explored outcomes among HIV+ KT recipients by antiretroviral therapy (ART) regimen. IMS pharmacy fills (1/1/01 - 10/1/12) were linked with SRTR data. 332 recipients with pre- and post-transplant fills were characterized by ART at time of transplant as protease inhibitor (PI) or non-PI based ART (88 PI vs. 244 non-PI). Cox proportional hazards models were adjusted for recipient and donor characteristics...
July 11, 2017: American Journal of Transplantation
https://www.readbyqxmd.com/read/28689878/systematic-review-of-antimicrobial-lock-therapy-for-prevention-of-central-line-associated-bloodstream-infections-in-adult-and-pediatric-cancer-patients
#8
REVIEW
LeAnn B Norris, Farah Kablaoui, Maggie K Brilhart, P Brandon Bookstaver
BACKGROUND: Central venous catheter (CVC) use is commonplace in cancer patients. Antimicrobial lock therapy (ALT), the instillation of a concentrated antimicrobial solution into the catheter lumen, is one method for preventing infection among CVCs. This systematic review discusses the effectiveness and safety of prophylactic ALT in cancer patients with CVCs. METHODS: A literature search was performed using the Medline database and Google Scholar from inception until April 2016...
July 6, 2017: International Journal of Antimicrobial Agents
https://www.readbyqxmd.com/read/28677778/wild%C3%A2-type-blocking-pcr-coupled-with-internal-competitive-amplified-fragment-improved-the-detection-of-rare-mutation-of-kras
#9
Jia Peng, Kun Wei, Xiang Zhao, Ke Yang, Huan Wang, Yang Zhang, Mei Guo, Jing He, Haiyan Wu, Yongchuan Li, Na Zhao, Qing Huang, Weiling Fu
Mutant KRAS proto‑oncogene GTPase (KRAS) serves an important role in predicting the development, diagnosis, treatment and efficacy of targeted drug therapies for colorectal cancer. To improve the detection efficacy of trace amount of mutant KRAS, the locked nucleic acid‑based method was modified in the present study. Internal competitive amplification fragments were used to improve the inhibition of wild‑type KRAS with a wild‑type blocking (WTB) probe and specifically amplify the trace amounts of mutant KRAS...
June 29, 2017: Molecular Medicine Reports
https://www.readbyqxmd.com/read/28675244/a-catheter-related-bloodstream-infection-caused-by-chryseobacterium-indologenes-successfully-treated-with-antibiotic-lock-rescue-therapy
#10
Marta Corbella, Micaela Brandolini, Patrizia Cambieri, Nunzia Decembrino, Michele Pagani, Andrea Bottazzi, Alba Muzzi, Marco Zecca, Bianca Mariani, Piero Marone
We report the case of a catheter-related bloodstream infection caused by Chryseobacterium indologenes, an uncommon and multi-resistant pathogen, in a pediatric patient with a long-term vascular access device placed for chemotherapy treatment. The infection was successfully treated with ciprofloxacin antibiotic-lock therapy. This is the first report on successful salvage of a long-term device colonized by multi-resistant Chryseobacterium indologenes.
July 4, 2017: New Microbiologica
https://www.readbyqxmd.com/read/28670496/resistance-to-cancer-chemotherapeutic-drugs-is-determined-by-pivotal-microrna-regulators
#11
Marta Geretto, Alessandra Pulliero, Camillo Rosano, Dinara Zhabayeva, Rakhmet Bersimbaev, Alberto Izzotti
Chemo-resistance, which is the main obstacle in cancer therapy, is caused by the onset of drug-resistant cells in the heterogeneous cell population in cancer tissues. MicroRNAs regulate gene expression at the post-transcriptional level, and they are involved in many different biological processes, including cell proliferation, differentiation, metabolism, stress response, and apoptosis. The aberrant expression of microRNAs plays a major pathogenic role from the early stages of the carcinogenesis process. Recently, microRNAs have been reported to play an important role in inducing resistance to anti-cancer drugs...
2017: American Journal of Cancer Research
https://www.readbyqxmd.com/read/28664750/preemptive-ethanol-lock-therapy-in-pediatric-hematology-oncology-patients-with-catheter-associated-bloodstream-infection-impact-on-length-of-stay-cost-and-catheter-salvage
#12
Eric McGrath, Wei Du, Madhvi Rajpurkar
BACKGROUND: Ethanol lock therapy (ELT) with systemic antimicrobial therapy is a promising therapy for catheter-related infection (CRI). The impact of ELT timing on treatment efficacy and costs is unknown. PROCEDURES: A prospective study was conducted in the Hematology/Oncology Unit at the Children's Hospital of Michigan. Patients with suspected CRI were randomized to Preemptive ELT arm or Rescue ELT arm after positive culture. RESULTS: Five cases in Preemptive arm and 9 in Rescue arm had a confirmed CRI...
June 1, 2017: Clinical Pediatrics
https://www.readbyqxmd.com/read/28660268/cancer-bioimprinting-and-cell-shape-recognition-for-diagnosis-and-targeted-treatment
#13
REVIEW
Jevan Medlock, Anupam A K Das, Leigh A Madden, David J Allsup, Vesselin N Paunov
Cancer incidence and mortality have both increased in the last decade and are predicted to continue to rise. Diagnosis and treatment of cancers are often hampered by the inability to specifically target neoplastic cells. Bioimprinting is a promising new approach to overcome shortfalls in cancer targeting. Highly specific recognition cavities can be made into polymer matrices to mimic lock-and-key actions seen in in vivo biological systems. Early studies concentrated on molecules and were inhibited by template size complexity...
June 29, 2017: Chemical Society Reviews
https://www.readbyqxmd.com/read/28658125/anterior-cervical-distraction-and-screw-elevating-pulling-reduction-for-traumatic-cervical-spine-fractures-and-dislocations-a-retrospective-analysis-of-86-cases
#14
Haoxi Li, Zhiyao Yong, Zhaoxiong Chen, Yufeng Huang, Zhoudan Lin, Desheng Wu
Treatment of cervical fracture and dislocation by improving the anterior cervical technique.Anterior cervical approach has been extensively used in treating cervical spine fractures and dislocations. However, when this approach is used in the treatment of locked facet joints, an unsatisfactory intraoperative reduction and prying reduction increases the risk of secondary spinal cord injury. Thus, herein, the cervical anterior approach was improved. With distractor and screw elevation therapy during surgery, the restoration rate is increased, and secondary injury to the spinal cord is avoided...
June 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28654545/impact-of-pretransplant-bridging-locoregional-therapy-for-patients-with-hepatocellular-carcinoma-within-milan-criteria-undergoing-liver-transplantation-analysis-of-3601-patients-from-the-us-multicenter-hcc-transplant-consortium
#15
Vatche G Agopian, Michael P Harlander-Locke, Richard M Ruiz, Goran B Klintmalm, Srinath Senguttuvan, Sander S Florman, Brandy Haydel, Maarouf Hoteit, Matthew H Levine, David D Lee, C Burcin Taner, Elizabeth C Verna, Karim J Halazun, Rita Abdelmessih, Amit D Tevar, Abhinav Humar, Federico Aucejo, William C Chapman, Neeta Vachharajani, Mindie H Nguyen, Marc L Melcher, Trevor L Nydam, Constance Mobley, R Mark Ghobrial, Beth Amundsen, James F Markmann, Alan N Langnas, Carol A Carney, Jennifer Berumen, Alan W Hemming, Debra L Sudan, Johnny C Hong, Joohyun Kim, Michael A Zimmerman, Abbas Rana, Michael L Kueht, Christopher M Jones, Thomas M Fishbein, Ronald W Busuttil
OBJECTIVE: To evaluate the effect of pretransplant bridging locoregional therapy (LRT) on hepatocellular carcinoma (HCC) recurrence and survival after liver transplantation (LT) in patients meeting Milan criteria (MC). SUMMARY BACKGROUND DATA: Pre-LT LRT mitigates tumor progression and waitlist dropout in HCC patients within MC, but data on its impact on post-LT recurrence and survival remain limited. METHODS: Recurrence-free survival and post-LT recurrence were compared among 3601 MC patients with and without bridging LRT utilizing competing risk Cox regression in consecutive patients from 20 US centers (2002-2013)...
June 26, 2017: Annals of Surgery
https://www.readbyqxmd.com/read/28638693/management-of-the-temporomandibular-joint-in-inflammatory-arthritis-involvement-of-surgical-procedures
#16
REVIEW
Rory C O'Connor, Fiona Fawthrop, Rami Salha, Andrew J Sidebottom
Many conditions may affect the temporomandibular joint (TMJ), but its incidence in individual joint diseases is low. However, inflammatory arthropathies, particularly rheumatoid and psoriatic arthritis and ankylosing spondylitis, appear to have a propensity for affecting the joint. Symptoms include pain, restriction in mouth opening, locking, and noises, which together can lead to significant impairment. Jaw rest, a soft diet, a bite splint, and medical therapy, including disease-modifying antirheumatic drugs (DMARDs) and simple analgesia, are the bedrock of initial treatment and will improve most symptoms in most patients...
June 2017: European Journal of Rheumatology
https://www.readbyqxmd.com/read/28623256/lna-dna-mixmer-based-antisense-oligonucleotides-correct-alternative-splicing-of-the%C3%A2-smn2-gene-and-restore-smn-protein-expression-in-type-1-sma-fibroblasts
#17
Aleksander Touznik, Rika Maruyama, Kana Hosoki, Yusuke Echigoya, Toshifumi Yokota
Spinal muscular atrophy (SMA) is an autosomal recessive disorder affecting motor neurons, and is currently the most frequent genetic cause of infant mortality. SMA is caused by a loss-of-function mutation in the survival motor neuron 1 (SMN1) gene. SMN2 is an SMN1 paralogue, but cannot compensate for the loss of SMN1 since exon 7 in SMN2 mRNA is excluded (spliced out) due to a single C-to-T nucleotide transition in the exon 7. One of the most promising strategies to treat SMA is antisense oligonucleotide (AON)-mediated therapy...
June 16, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28577210/-distal-femoral-osteotomy-using-a-lateral-opening-wedge-technique
#18
M J Feucht, J Mehl, P Forkel, A B Imhoff, S Hinterwimmer
OBJECTIVE: To shift the weight-bearing axis of the lower limb medially by opening a lateral-based metaphyseal osteotomy at the distal femur. INDICATIONS: Femoral-based valgus malalignment and symptomatic lateral unicompartimental osteoarthritis, lateral hyperpression syndrome, cartilage therapy of the lateral compartment, lateral meniscal replacement/transplantation, medial instability with valgus thrust, reconstruction of the medial collateral ligament, patellar instability and/or maltracking...
June 2, 2017: Operative Orthopädie und Traumatologie
https://www.readbyqxmd.com/read/28576557/a-prospective-double-blind-randomized-controlled-study-on-the-use-of-ethanol-locks-in-hpn-patients
#19
Bradley R Salonen, Sara L Bonnes, Nishanth Vallumsetla, Jithinraj Edakkanambeth Varayil, Manpreet S Mundi, Ryan T Hurt
BACKGROUND & AIMS: Ethanol lock therapy (ELT) has been shown to reduce the rate of catheter-related bloodstream infection (CRBSI) in high-risk home parenteral nutrition (HPN) patients. The aim of this study was to determine whether ELT therapy for all patients newly started on HPN would reduce the incidence of CRBSI. METHODS: This study was a prospective, double-blind, randomized controlled trial that was carried out from July 2014 to April 2016. The study participants were patients newly started on HPN, and they were randomly assigned to either treatment with ELT or our current standard of care with saline heparin locks...
May 17, 2017: Clinical Nutrition: Official Journal of the European Society of Parenteral and Enteral Nutrition
https://www.readbyqxmd.com/read/28575416/toxic-drug-induced-liver-failure-during-therapy-of-rheumatoid-arthritis-with-tocilizumab-subcutaneously-a-case-report
#20
Friedrich Anger, Armin Wiegering, Johanna Wagner, Johan Lock, Johannes Baur, Lukas Haug, Marc Schmalzing, Andreas Geier, Stefan Löb, Ingo Klein
No abstract text is available yet for this article.
June 1, 2017: Rheumatology
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