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Precision therapy

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https://www.readbyqxmd.com/read/27926889/precision-medicine-diabetes-and-the-u-s-food-and-drug-administration
#1
Robert J Meyer
The U.S. Food and Drug Administration (FDA) has long sought to achieve the broader use of personalized medicine, which is better targeting of FDA-approved therapies through incorporating precise knowledge of a patient's underlying condition to therapies optimally chosen to match those needs. While some strides have been made in precision medicine-particularly in oncology and rare genetic diseases-most of the standard general medicine indications have yet to realize the benefits of precision-guided therapies...
November 2016: Diabetes Care
https://www.readbyqxmd.com/read/27924634/the-promise-of-circulating-tumor-cells-for-precision-cancer-therapy
#2
William L Hwang, Katie L Hwang, David T Miyamoto
The rapidly growing array of therapeutic options in cancer requires informative biomarkers to guide the rational selection and precision application of appropriate therapies. Circulating biomarkers such as circulating tumor cells have immense potential as noninvasive, serial 'liquid biopsies' that may be more representative of the complete spectrum of a patient's individual malignancy than spatially and temporally restricted tumor biopsies. In this review, we discuss the current state-of-the-art in the isolation and molecular characterization of circulating tumor cells as well as their utility in a wide range of clinical applications such as prognostics, treatment monitoring and identification of novel therapeutic targets and resistance mechanisms to enable real-time adjustments in the clinical management of cancer...
December 7, 2016: Biomarkers in Medicine
https://www.readbyqxmd.com/read/27924064/toward-the-use-of-precision-medicine-for-the-treatment-of-head-and-neck-squamous-cell-carcinoma
#3
REVIEW
Wang Gong, Yandi Xiao, Zihao Wei, Yao Yuan, Min Qiu, Chongkui Sun, Xin Zeng, Xinhua Liang, Mingye Feng, Qianming Chen
Precision medicine is a new strategy that aims at preventing and treating human diseases by focusing on individual variations in people's genes, environment and lifestyle. Precision medicine has been used for cancer diagnosis and treatment and shows evident clinical efficacy. Rapid developments in molecular biology, genetics and sequencing technologies, as well as computational technology, has enabled the establishment of "big data", such as the Human Genome Project, which provides a basis for precision medicine...
December 4, 2016: Oncotarget
https://www.readbyqxmd.com/read/27922722/topiramate-versus-carbamazepine-monotherapy-for-epilepsy-an-individual-participant-data-review
#4
REVIEW
Sarah J Nolan, Maria Sudell, Catrin Tudur Smith, Anthony G Marson
BACKGROUND: Epilepsy is a common neurological condition in which abnormal electrical discharges from the brain cause recurrent unprovoked seizures. It is believed that with effective drug treatment, up to 70% of individuals with active epilepsy have the potential to become seizure-free and go into long-term remission shortly after starting drug therapy, the majority of which may be able to achieve remission with a single antiepileptic drug (AED).The correct choice of first-line antiepileptic therapy for individuals with newly diagnosed seizures is of great importance...
December 6, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27922501/an-overview-of-congenital-myopathies
#5
Jean K Mah, Jeffrey T Joseph
PURPOSE OF REVIEW: This article uses a case-based approach to highlight the clinical features as well as recent advances in molecular genetics, muscle imaging, and pathophysiology of the congenital myopathies. RECENT FINDINGS: Congenital myopathies refer to a heterogeneous group of genetic neuromuscular disorders characterized by early-onset muscle weakness, hypotonia, and developmental delay. Congenital myopathies are further classified into core myopathies, centronuclear myopathies, nemaline myopathies, and congenital fiber-type disproportion based on the key pathologic features found in muscle biopsies...
December 2016: Continuum: Lifelong Learning in Neurology
https://www.readbyqxmd.com/read/27922044/from-tumour-heterogeneity-to-advances-in-precision-treatment-of-colorectal-cancer
#6
REVIEW
Cornelis J A Punt, Miriam Koopman, Louis Vermeulen
In recent years, the high heterogeneity of colorectal cancer (CRC) has become evident. Hence, biomarkers need to be developed that enable the stratification of patients with CRC into different prognostic subgroups and in relation to response to therapies, according to the distinctive tumour biology. Currently, only RAS-mutation status is used routinely as a negative predictive marker to avoid treatment with anti-EGFR agents in patients with metastatic CRC, and mismatch-repair status can guide the use of adjuvant chemotherapy in patients with early stage colon cancer...
December 6, 2016: Nature Reviews. Clinical Oncology
https://www.readbyqxmd.com/read/27920899/diagnosing-clean-margins-through-raman-spectroscopy-in-human-and-animal-mammary-tumour-surgery-a-short-review
#7
REVIEW
I A Birtoiu, C Rizea, D Togoe, R M Munteanu, C Micsa, M I Rusu, M Tautan, L Braic, L O Scoicaru, A Parau, N D Becherescu-Barbu, M V Udrea, A Tonetto, R Notonier, C E A Grigorescu
Breast cancer frequency in human and other mammal female populations has worryingly increased lately. The acute necessity for taxonomy of the aetiological factors along with seeking for new diagnostic tools and therapy procedures aimed at reducing mortality have yielded in an intense research effort worldwide. Surgery is a regular method to counteract extensive development of breast cancer and prevent metastases provided that negative surgical margins are achieved. This highly technical challenge requires fast, extremely sensitive and selective discrimination between malignant and benign tissues even down to molecular level...
December 6, 2016: Interface Focus
https://www.readbyqxmd.com/read/27920633/lesch-nyhan-syndrome-models-theories-and-therapies
#8
REVIEW
Scott Bell, Ilaria Kolobova, Liam Crapper, Carl Ernst
Lesch-Nyhan syndrome (LNS) is a rare X-linked disorder caused by mutations in HPRT1, an important enzyme in the purine salvage pathway. Symptoms of LNS include dystonia, gout, intellectual disability, and self-mutilation. Despite having been characterized over 50 years ago, it remains unclear precisely how deficits in hypoxanthine and guanine recycling can lead to such a profound neurological phenotype. Several studies have proposed different hypotheses regarding the etiology of this disease, and several treatments have been tried in patients, though none have led to a satisfactory explanation of the disease...
November 2016: Molecular Syndromology
https://www.readbyqxmd.com/read/27920501/three-generations-of-epidermal-growth-factor-receptor-tyrosine-kinase-inhibitors-developed-to-revolutionize-the-therapy-of-lung-cancer
#9
REVIEW
Haijun Zhang
Lung cancer, ~80%-85% of which is non-small-cell lung cancer (NSCLC), is the leading cause of cancer-related mortality worldwide. Sensitizing mutations in epidermal growth factor receptor (EGFR) gene (EGFRm(+)), such as exon 19 deletions and exon 21 L858R point mutations, are the most important drivers in NSCLC patients. In this respect, small-molecule EGFR tyrosine kinase inhibitors (TKIs) have been designed and developed, which launched the era of targeted, personalized and precise medicine for lung cancer...
2016: Drug Design, Development and Therapy
https://www.readbyqxmd.com/read/27919119/-osteochondritis-dissecans-of-the-talus-a-critical-review
#10
J Bruns, C R Habermann, J P Petersen
The etiology remains unclear. Mechanical factors seem to play an important role. Most histologies report clefts between the cartilage and bone and necrotic areas. Several classifications have been published based on observations from imaging techniques or on intraoperative findings. Clinical symptoms are unspecific. Imaging techniques are of great importance: X-rays of the ankle joint enable a quick diagnostic overview. MRT is the imaging-technique of choice for diagnosing OCDT-lesions. Regarding treatment, a tremendous number of retrospective publications exist...
April 2016: Zeitschrift Für Orthopädie und Unfallchirurgie
https://www.readbyqxmd.com/read/27917695/nanostring-a-novel-digital-color-coded-barcode-technology-current-and-future-applications-in-molecular-diagnostics
#11
Hin-Fung Tsang, Vivian Weiwen Xue, Su-Pin Koh, Ya-Ming Chiu, Lawrence Po-Wah Ng, Sze-Chuen Cesar Wong
Formalin-fixed, paraffin-embedded (FFPE) tissue sample is a gold mine of resources for molecular diagnosis and retrospective clinical studies. Although molecular technologies have expanded the range of mutations identified in FFPE samples, the applications of existing technologies are limited by the low nucleic acids yield and poor extraction quality. As a result, the routine clinical applications of molecular diagnosis using FFPE samples has been associated with many practical challenges. NanoString technologies utilize a novel digital color-coded barcode technology based on direct multiplexed measurement of gene expression and offer high levels of precision and sensitivity...
December 3, 2016: Expert Review of Molecular Diagnostics
https://www.readbyqxmd.com/read/27916607/current-status-of-biomarker-and-targeted-nanoparticle-development-the-precision-oncology-approach-for-pancreatic-cancer-therapy
#12
Lei Zhu, Charles Staley, David Kooby, Bassel El-Rays, Hui Mao, Lily Yang
Pancreatic cancer remains one of the major causes of cancer-related mortality. The majority of pancreatic cancer patients are diagnosed at the advanced stage with unresectable and drug resistant tumors. The new treatments with the combination of chemotherapy, molecular targeted therapy, and immunotherapy have shown modest effects on therapeutic efficacy and survival of the patients. Therefore, there is an urgent need to develop effective therapeutic approaches targeting highly heterogeneous pancreatic cancer cells and tumor microenvironments...
December 1, 2016: Cancer Letters
https://www.readbyqxmd.com/read/27916286/relating-therapy-for-distressing-auditory-hallucinations-a-pilot-randomized-controlled-trial
#13
Mark Hayward, Anna-Marie Jones, Leanne Bogen-Johnston, Neil Thomas, Clara Strauss
Auditory hallucinations (AH) are a common and distressing experience and patients report distress reduction to be a priority. Relating Therapy adopts a symptom-specific and mechanism focused approach to the reduction of AH distress. We conducted this single-blind, pragmatic, parallel groups, superiority pilot RCT within a single mental health centre in the UK. Patients (18+years) with persistent and distressing AH, irrespective of diagnosis were randomly allocated to receive either Relating Therapy and Treatment-as-usual (RT) or Treatment as-usual alone (TAU)...
December 1, 2016: Schizophrenia Research
https://www.readbyqxmd.com/read/27915475/how-precisely-can-prostate-cancer-be-managed
#14
REVIEW
Liyan Zhuang, Matthew T Johnson
Progress has been made in applying genetic information to disease management in the postgenomic era, and precision medicine is emerging in prostate cancer management. The prostate health index, the 4-kallikrein (4K) score, and the PCA3, TMPRSS2- ERG, and Prostarix tests have potential for refining prostate cancer screening in conjunction with traditional prostate-specific antigen testing. The Confirm MDx and PCA3 tests have shown promise in identifying men who need be rebiopsied after a primary negative biopsy...
November 2016: International Neurourology Journal
https://www.readbyqxmd.com/read/27915328/critical-care-nephrology-a-multidisciplinary-approach
#15
Lilia Rizo-Topete, Claudio Ronco
Acute kidney injury (AKI) is a serious medical condition affecting millions of people. Patients in intensive care unit (ICU) who develop AKI have increased morbidity and mortality, prolonged length of stay in ICU and hospital and increased costs, especially when they require renal replacement therapy. In the latter case, morbidity and mortality increase further. In order to meet the needs of the critically ill patients, a multidisciplinary care team is required, combining the efforts of physicians and nurses from different disciplines as well as nephrologists and intensivists...
December 3, 2016: Blood Purification
https://www.readbyqxmd.com/read/27915035/hyperinsulinaemic-hypoglycaemia-in-children-and-adults
#16
REVIEW
Pratik Shah, Sofia A Rahman, Huseyin Demirbilek, Maria Güemes, Khalid Hussain
Pancreatic β cells are functionally programmed to release insulin in response to changes in plasma glucose concentration. Insulin secretion is precisely regulated so that, under normal physiological conditions, fasting plasma glucose concentrations are kept within a narrow range of 3·5-5·5 mmol/L. In hyperinsulinaemic hypoglycaemia, insulin secretion becomes dysregulated (ie, uncoupled from glucose metabolism) so that insulin secretion persists in the presence of low plasma glucose concentrations. Hyperinsulinaemic hypoglycaemia is the most common cause of severe and persistent hypoglycaemia in neonates and children...
November 30, 2016: Lancet Diabetes & Endocrinology
https://www.readbyqxmd.com/read/27915023/anisotropic-noble-metal-nanoparticles-synthesis-surface-functionalization-and-applications-in-biosensing-bioimaging-drug-delivery-and-theranostics
#17
REVIEW
Gokul Paramasivam, Namitharan Kayambu, Arul Maximus Rabel, Ashok K Sundramoorthy, Anandhakumar Sundaramurthy
Anisotropic nanoparticles have fascinated scientists and engineering communities for over a century because of their unique physical and chemical properties. In recent years, continuous advances in design and fabrication of anisotropic nanoparticles have opened new avenues for application in various areas of biology, chemistry and physics. Anisotropic nanoparticles have the plasmon absorption in the visible as well as near-infrared (NIR) region, which enable them to be used for crucial applications such as biological imaging, medical diagnostics and therapy ("theranostics")...
November 30, 2016: Acta Biomaterialia
https://www.readbyqxmd.com/read/27913529/ph-like-acute-lymphoblastic-leukemia
#18
Thai Hoa Tran, Mignon L Loh
Philadelphia chromosome-like acute lymphoblastic leukemia (Ph-like ALL) is a newly identified high-risk (HR) B-lineage ALL subtype, accounting for ∼15% of children with National Cancer Institute-defined HR B-ALL. It occurs more frequently in adolescents and adults, having been reported in as much as 27% of young adults with ALL between 21 and 39 years of age. It exhibits adverse clinical features, confers a poor prognosis, and harbors a diverse range of genetic alterations that activate cytokine receptor genes and kinase signaling pathways, making it amenable to treatment with tyrosine kinase inhibitor (TKI) therapy...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27913517/treatment-of-low-risk-myelodysplastic-syndromes
#19
Valeria Santini
The majority of myelodysplastic syndrome (MDS) patients belong to the International Prognostic Scoring System (IPSS) and IPSS-revised (IPSS-R) lower-risk categories. Their precise diagnostics and prognostic stratification is often a challenge, but may ensure the optimization of therapy. The availability of diverse treatment options has significantly improved the quality of life and survival of this group of patients. Anemia is the most relevant cytopenia in terms of frequency and symptoms in lower-risk MDS, and may be treated successfully with erythropoietic stimulating agents, provided a careful selection is performed on the basis of IPSS-R, endogenous erythropoietin levels, and transfusion independence...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27913491/prognostic-factors-in-follicular-lymphoma-new-tools-to-personalize-risk
#20
Carla Casulo
Follicular lymphoma (FL) is the most common indolent lymphoma, and it has a long median overall survival (OS). However, the recent discovery of clinical and biological prognostic biomarkers in FL is shedding light on FL heterogeneity and the need for a precise and risk-stratified individual approach at diagnosis and relapse. Many FL patients who are asymptomatic with indolent disease can be vulnerable to the toxicity, emotional distress, and financial burden of overtreatment. Yet a subset of FL patients develop chemoresistance to standard chemoimmunotherapy, experience transformation to aggressive lymphoma and rapid progression, and represent the population most in need of novel therapies and curative approaches...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
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