How we treat thrombotic thrombocytopenic purpura: Results of a Canadian TTP practice survey.

J Clin Apher. 2016 Jul 31;

Authors: Patriquin CJ, Clark WF, Pavenski K, Arnold DM, Rock G, Foley SR, Canadian Apheresis Group

Abstract
BACKGROUND: Thrombotic thrombocytopenic purpura (TTP) is a rare disease with 90% mortality if untreated. Since the Canadian Apheresis Group (CAG) trial showed greater survival with therapeutic plasma exchange (TPE) versus plasma infusion, there has been widespread adoption of TPE. Beyond TPE, there is significant practice variation. To characterize this, we developed a survey sent to physicians who might be directly involved in TTP management.
METHODS: The survey was sent to CAG members as well as hematologists and nephrologists nationwide and addressed areas of controversy or recognized practice heterogeneity. Descriptive statistics were used to summarize responses, and the χ(2) test was used to compare respondents who were and were not CAG physicians. We also compared responses by estimated frequency of TTP cases per year.
RESULTS: The CAG response rate was 31% (13 of 42). The survey was sent to 665 non-CAG physicians, of whom 41 responded (6.1%). Though not statistically different, CAG and non-CAG respondents varied regarding use of corticosteroids, aspirin, and venous thromboembolism (VTE) prophylaxis. Significant differences were found between CAG and non-CAG groups regarding cryosupernatant as fluid choice (69.2% vs. 22.5%, P = .004) and the use of TPE tapering (84.6% vs. 51.3%, P = .034), respectively.
CONCLUSION: TTP treatment is variable across centres in Canada. Areas of significant variation include the choice of replacement fluid for TPE and whether or not and how to taper TPE. Our survey highlights the practice heterogeneity that exists and identifies areas where more evidence is needed and perhaps where trials should be performed.

PMID: 27476033 [PubMed - as supplied by publisher]

Neonatal Lupus: What We Have Learned and Current Approaches to Care.

Curr Rheumatol Rep. 2016 Sep;18(9):60

Authors: Klein-Gitelman MS

Abstract
Neonatal lupus results from the passive transfer of autoantibodies; however, this transfer is not sufficient to cause disease. This article reviews clinical presentation with a focus on autoimmune-mediated congenital heart disease. Recent data looking for additional disease mechanisms and biomarkers as well as latest information on interventions will be reviewed. Our understanding of this rare disease is often dependent on patient participation in disease registries and biorepositories. Future participation in registries including descriptive as well as biophysical data is critical to our knowledge.

PMID: 27475948 [PubMed - as supplied by publisher]

Impact of rare diseases in oral health.

Med Oral Patol Oral Cir Bucal. 2016 Jul 31;:0

Authors: Molina-García A, Castellanos-Cosano L, Machuca-Portillo G, Posada-de la Paz M

Abstract
BACKGROUND: Rare diseases (RD) are those that present a lower prevalence than 5 cases per 10.000 population. The main objective of this review was to study the effect on oral health in rare diseases, while the secondary objective of the study is theme upgrade.
MATERIAL AND METHODS: Comparative observational case-control studies were analysed and a systematic review was conducted in PubMed. Each rare disease listed on the statistical data record of the Health Portal of the Ministry of Equality, Health and Social Policies Board of Andalusia was associated with "oral health". The variables studied included dental, oral mucosa and occlusion alterations, oral pathologies (caries, periodontal disease) and other alterations (mouth breathing, parafunctional habits, etc). A bias analysis of the variable caries was conducted.
RESULTS: Six RD were selected through our inclusion and exclusion criteria (hypogammaglobulinemia, Rett syndrome, Marfan syndrome, Prader-Willi syndrome, cystic fibrosis and Cri du chat syndrome) in a total of 8 publications, of which four trials were classified as high risk of bias and one of them as medium risk. There were not trials with low risk of bias.
CONCLUSIONS: The main statistically significant differences found by Syndrome compared to a control group were in Hypogammaglobulinemia with a greater tendency to enamel hypoplasia and dry mouth. The Rett syndrome had, as well, a greater tendency to an anterior open bite, ogival palate, bruxism, mouth breathing and tongue thrusting. Prader-Willi syndrome had a tendency of dental erosion, and Cri du chat syndrome showed a higher association to Tannerella forsythia.

PMID: 27475682 [PubMed - as supplied by publisher]

Orofacial manifestations of scleroderma. A literature review.

Rev Stomatol Chir Maxillofac Chir Orale. 2016 Jul 27;

Authors: Hadj Said M, Foletti JM, Graillon N, Guyot L, Chossegros C

Abstract
INTRODUCTION: Scleroderma is a rare disease of the connective tissue (50 to 200 patients/1 million people; 60,000 patients in France). We conducted a literature review about the orofacial manifestations of scleroderma that have been little studied.
MATERIAL AND METHODS: The 45 articles found in 6 different databases by using the keywords "scleroderma", "systemic sclerosis", "oral medicine", "face" and published between 1944 and 2016 were selected, for a total of 328 patients.
RESULTS: A total of 1187 orofacial manifestations of scleroderma were identified, occurring mainly in women (84.5%) with a mean age of 40.2 years, 10 years on average after the first manifestation of the disease. The main ones were limitation of mouth opening (69.8%), widening of the periodontal ligament (67.3%), xerostomia (63.4%), telangiectasia (36.2%) and bone lesions (34.5%). Dental root resorptions, pulp and nose calcifications were also reported but with no evident link with scleroderma.
DISCUSSION: Orofacial manifestations of scleroderma are probably more common than reported. They mostly affect women with a mean age of 40. The most common oral manifestations are limitation of mouth opening, widening of the periodontal ligament and xerostomia. Because of the handicap they may be responsible for, these manifestations must be detected early in order to prevent from functional impairments and from dental and periodontal lesions.

PMID: 27475503 [PubMed - as supplied by publisher]

Ivacaftor and symptoms of extra-oesophageal reflux in patients with cystic fibrosis and G551D mutation.

J Cyst Fibros. 2016 Jul 27;

Authors: Zeybel GL, Pearson JP, Krishnan A, Bourke SJ, Doe S, Anderson A, Faruqi S, Morice AH, Jones R, McDonnell M, Zeybel M, Dettmar PW, Brodlie M, Ward C

Abstract
BACKGROUND: Extra-oesophageal reflux (EOR) may lead to microaspiration in patients with cystic fibrosis (CF), a probable cause of deteriorating lung function. Successful clinical trials of ivacaftor highlight opportunities to understand EOR in a real world study.
METHODS: Data from 12 patients with CF and the G551D mutation prescribed ivacaftor (150mg bd) was collected at baseline, 6, 26 and 52weeks. The changes in symptoms of EOR were assessed by questionnaire (reflux symptom index (RSI) and Hull airway reflux questionnaire (HARQ)).
RESULTS: Six patients presented EOR at baseline (RSI >13; median 13; range 2-29) and 5 presented airway reflux (HARQ >13; median 12; range 3 to 33). Treatment with ivacaftor was associated with a significant reduction of EOR symptoms (P<0∙04 versus baseline) denoted by the reflux symptom index and Hull airway reflux questionnaire.
CONCLUSION: Ivacaftor treatment was beneficial for patients with symptoms of EOR, thought to be a precursor to microaspiration.

PMID: 27475719 [PubMed - as supplied by publisher]

Impact of rare diseases in oral health.

Med Oral Patol Oral Cir Bucal. 2016 Jul 31;:0

Authors: Molina-García A, Castellanos-Cosano L, Machuca-Portillo G, Posada-de la Paz M

Abstract
BACKGROUND: Rare diseases (RD) are those that present a lower prevalence than 5 cases per 10.000 population. The main objective of this review was to study the effect on oral health in rare diseases, while the secondary objective of the study is theme upgrade.
MATERIAL AND METHODS: Comparative observational case-control studies were analysed and a systematic review was conducted in PubMed. Each rare disease listed on the statistical data record of the Health Portal of the Ministry of Equality, Health and Social Policies Board of Andalusia was associated with "oral health". The variables studied included dental, oral mucosa and occlusion alterations, oral pathologies (caries, periodontal disease) and other alterations (mouth breathing, parafunctional habits, etc). A bias analysis of the variable caries was conducted.
RESULTS: Six RD were selected through our inclusion and exclusion criteria (hypogammaglobulinemia, Rett syndrome, Marfan syndrome, Prader-Willi syndrome, cystic fibrosis and Cri du chat syndrome) in a total of 8 publications, of which four trials were classified as high risk of bias and one of them as medium risk. There were not trials with low risk of bias.
CONCLUSIONS: The main statistically significant differences found by Syndrome compared to a control group were in Hypogammaglobulinemia with a greater tendency to enamel hypoplasia and dry mouth. The Rett syndrome had, as well, a greater tendency to an anterior open bite, ogival palate, bruxism, mouth breathing and tongue thrusting. Prader-Willi syndrome had a tendency of dental erosion, and Cri du chat syndrome showed a higher association to Tannerella forsythia.

PMID: 27475682 [PubMed - as supplied by publisher]

Cystic Fibrosis Papers of the Year 2015.

Paediatr Respir Rev. 2016 Jun 15;

Authors: Doull I

Abstract
Studies published in the last year have expanded our knowledge of potential disease modifying agents in the treatment of class II, III and IV CFTR mutations, and included the first report of an efficacious gene therapy for CF. There is also an important message on increasing use of conventional chronic therapies even in milder disease, and the pernicious effect of chronic infection on pulmonary function.

PMID: 27475293 [PubMed - as supplied by publisher]

ROHHAD syndrome and evolution of sleep disordered breathing.

Orphanet J Rare Dis. 2016;11(1):106

Authors: Reppucci D, Hamilton J, Yeh EA, Katz S, Al-Saleh S, Narang I

Abstract
BACKGROUND: Rapid-onset obesity with hypothalamic dysfunction, hypoventilation and autonomic dysregulation (ROHHAD) is a rare disease with a high mortality rate. Although nocturnal hypoventilation (NH) is central to ROHHAD, the evolution of sleep disordered breathing (SDB) is not well studied. The aim of the study was to assess early manifestations of SDB and their evolution in ROHHAD syndrome.
METHODS: Retrospective study of children with ROHHAD at two Canadian centers. All children with suspected ROHHAD at presentation underwent polysomnography (PSG) to screen for nocturnal hypoventilation. PSG findings at baseline and follow-up were collected. Interventions and diagnostic test results were recorded.
RESULTS: Six children were included. The median age of rapid onset obesity and nocturnal hypoventilation (NH) was 3.5 and 7.2 years respectively. On initial screening for ROHHAD 4/6 (66.7 %) children had obstructive sleep apnea (OSA), 1/6 (16.7 %) had NH and 1/6 (16.7 %) had both OSA and NH. Follow up PSGs were performed in 5/6 children as one child died following a cardiorespiratory arrest. All children at follow up had NH and required non-invasive positive pressure ventilation. Additionally, 3/6 (50 %) children demonstrated irregular breathing patterns during wakefulness.
CONCLUSIONS: Children with ROHHAD may initially present with OSA and only develop NH later as well as dysregulation of breathing during wakefulness. The recognition of the spectrum of respiratory abnormalities at presentation and over time may be important in raising the index of suspicion of ROHHAD. Early recognition and targeted therapeutic interventions may limit morbidity and mortality associated with ROHHAD.

PMID: 27473663 [PubMed - as supplied by publisher]

Familial Mediterranean fever is no longer a rare disease in Japan.

Arthritis Res Ther. 2016;18(1):175

Authors: Migita K, Izumi Y, Jiuchi Y, Iwanaga N, Kawahara C, Agematsu K, Yachie A, Masumoto J, Fujikawa K, Yamasaki S, Nakamura T, Ubara Y, Koga T, Nakashima Y, Shimizu T, Umeda M, Nonaka F, Yasunami M, Eguchi K, Yoshiura KI, Kawakami A

Abstract
BACKGROUND: The aim of this study was to evaluate the clinical manifestations and prevalence of familial Mediterranean fever (FMF) in Japanese patients with unexplained fever and rheumatic manifestations.
METHODS: We enrolled 601 patients with unexplained fever or suspected FMF throughout Japan between 2009 and 2015. Patients were divided into three groups according to Tel Hashomer criteria: sure FMF, probable FMF, and non-FMF patients, including definitive rheumatic diseases. Mutation detection in exons 1, 2, 3, and 10 of the FMF gene MEFV was performed by direct sequencing.
RESULTS: A total of 192 patients (31.9 %) were diagnosed with FMF according to FMF diagnostic criteria. These could be divided into sure FMF (56.3 %, n = 108) and probable FMF (43.7 %, n = 84) patients. Fever, abdominal symptoms, and thoracic symptoms were significantly more common in FMF than non-FMF patients. Among FMF patients, 26 (13.5 %) had concomitant rheumatic diseases. Most FMF patients (94.3 %, 181/192) carried at least one MEFV mutation. Allele frequencies of M694I (13.5 % vs 0 %) and E148Q (39.1 % vs 24.8 %) mutations were significantly higher in FMF compared with healthy subjects. Allele frequencies of common MEFV mutations in FMF patients were M694I (13.5 %), P369S (8.6 %), R408Q (8.1 %), G304R (2.9 %), R202Q (4.4 %), E148Q (39.1 %), L110P (11.7 %), and E84K (3.1 %). Patients with a sure FMF phenotype had a higher frequency of MEFV exon 10 mutation (M694I) and a lower frequency of MEFV exon 3 mutations (P369S, R408Q) compared with those with a probable FMF phenotype.
CONCLUSION: The high prevalence of FMF in Japanese patients with unexplained fever was confirmed in the present study. FMF should be suspected in cases of unexplained fever or non-specific rheumatic manifestations, and mutational analysis of MEFV could be useful to predict the clinical phenotypes of FMF in Japan.

PMID: 27473114 [PubMed - as supplied by publisher]

Context-Awareness Based Personalized Recommendation of Anti-Hypertension Drugs.

J Med Syst. 2016 Sep;40(9):202

Authors: Chen D, Jin D, Goh TT, Li N, Wei L

Abstract
The World Health Organization estimates that almost one-third of the world's adult population are suffering from hypertension which has gradually become a "silent killer". Due to the varieties of anti-hypertensive drugs, patients are interested in how these drugs can be selected to match their respective conditions. This study provides a personalized recommendation service system of anti-hypertensive drugs based on context-awareness and designs a context ontology framework of the service. In addition, this paper introduces a Semantic Web Rule Language (SWRL)-based rule to provide high-level context reasoning and information recommendation and to overcome the limitation of ontology reasoning. To make the information recommendation of the drugs more personalized, this study also devises three categories of information recommendation rules that match different priority levels and uses a ranking algorithm to optimize the recommendation. The experiment conducted shows that combining the anti-hypertensive drugs personalized recommendation service context ontology (HyRCO) with the optimized rule reasoning can achieve a higher-quality personalized drug recommendation service. Accordingly this exploratory study of the personalized recommendation service for hypertensive drugs and its method can be easily adopted for other diseases.

PMID: 27473866 [PubMed - as supplied by publisher]

Trafficking and Function of the Cystic Fibrosis Transmembrane Conductance Regulator: A Complex Network of Post-Translational Modifications.

Am J Physiol Lung Cell Mol Physiol. 2016 Jul 29;:ajplung.00431.2015

Authors: McClure ML, Barnes S, Brodsky JL, Sorscher EJ

Abstract
Post-translational modifications add diversity to protein function. Throughout its life cycle, the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) undergoes numerous covalent post-translational modifications (PTMs), including glycosylation, ubiquitination, sumoylation, phosphorylation, and palmitoylation. These modifications regulate key steps during protein biogenesis, such as protein folding, trafficking, stability, function, and association with protein partners, and therefore may serve as targets for therapeutic manipulation. More generally, an improved understanding of molecular mechanisms that underlie CFTR PTMs may suggest novel treatment strategies for CF and perhaps other protein conformational diseases. This review provides a comprehensive summary of co- and post-translational CFTR modifications and their significance with regard to protein biogenesis.

PMID: 27474090 [PubMed - as supplied by publisher]

The effects of ivacaftor on CF fatty acid metabolism: An analysis from the GOAL study.

J Cyst Fibros. 2016 Jul 26;

Authors: O'Connor MG, Seegmiller A

Abstract
BACKGROUND: Ivacaftor has produced significant improvement in certain individuals with cystic fibrosis (CF), though the full metabolic effects of treatment remain unknown. Abnormalities in fatty acid metabolism have previously been shown to be a characteristic of CFTR dysfunction. We hypothesized that as a reflection of this clinical improvement, ivacaftor would improve plasma fatty acid levels and decrease urine prostaglandin E metabolite levels.
METHODS: This study analyzed plasma fatty acid levels and urine prostaglandin E metabolites (PGE-M) in 40 subjects with CF participating in the G551D observational (GOAL) study who demonstrated response to the medication by a significant decrease in sweat Cl levels. Paired samples were analyzed before and after 6months of ivacaftor treatment.
RESULTS: Linoleic acid and docosahexaenoic acid levels, which are typically low in individuals with CF, did not significantly increase with ivacaftor treatment. However, arachidonic acid levels did decrease with ivacaftor treatment and there was a significant decrease in the arachidonic acid metabolite PGE-M as measured in the urine [median: before treatment 17.03ng/mg Cr; after treatment 9.06ng/mg Cr; p<0.001]. Furthermore, there were fatty acid age differences observed, including pediatric participants having significantly greater linoleic acid levels at baseline.
CONCLUSION: Ivacaftor reduces inflammatory PGE without fully correcting the plasma fatty acid abnormalities of CF. Age-related differences in fatty acid levels were observed, that may be a result of other clinical factors, such as diet, clinical care, or drug response.

PMID: 27473897 [PubMed - as supplied by publisher]

Synthesis and electrochemical detection of a thiazolyl-indole natural product isolated from the nosocomial pathogen Pseudomonas aeruginosa.

Anal Bioanal Chem. 2016 Jul 29;

Authors: Buzid A, Muimhneacháin EÓ, Reen FJ, Hayes PE, Pardo LM, Shang F, O'Gara F, Sperry J, Luong JH, Glennon JD, McGlacken GP

Abstract
Pseudomonas aeruginosa is a Gram-negative opportunistic pathogen, capable of surviving in a broad range of natural environments and quickly acquiring resistance. It is associated with hospital-acquired infections, particularly in patients with compromised immunity, and is the primary cause of morbidity and mortality in cystic fibrosis (CF) patients. P. aeruginosa is also of nosocomial importance on dairy farms and veterinary hospitals, where it is a key morbidity factor in bovine mastitis. P. aeruginosa uses a cell-cell communication system consisting of signalling molecules to coordinate bacterial secondary metabolites, biofilm formation, and virulence. Simple and sensitive methods for the detection of biomolecules as indicators of P. aeruginosa infection would be of great clinical importance. Here, we report the synthesis of the P. aeruginosa natural product, barakacin, which was recently isolated from the bovine ruminal strain ZIO. A simple and sensitive electrochemical method was used for barakacin detection using a boron-doped diamond (BDD) and glassy carbon (GC) electrodes, based on cyclic voltammetry (CV) and differential pulse voltammetry (DPV). The influence of electrolyte pH on the peak potential and peak currents was also investigated. At pH 2.0, the peak current was linearly dependent on barakacin concentration (in the range used, 1-10 μM), with correlation coefficients greater than 0.98 on both electrodes. The detection limit (S/N = 3) on the BDD electrode was 100-fold lower than that obtained on the GC electrode. The optimized method using the BDD electrode was extended to bovine (cow feces) and human (sputum of a CF patient) samples. Spiked barakacin was easily detected in these matrices at a limit of 0.5 and 0.05 μM, respectively. Graphical abstract Electrochemical detection of barakacin.

PMID: 27473426 [PubMed - as supplied by publisher]

Pathogen-induced secretory diarrhea and its prevention.

Eur J Clin Microbiol Infect Dis. 2016 Jul 29;

Authors: Anand S, Mandal S, Patil P, Tomar SK

Abstract
Secretory diarrhea is a historically known serious health implication around the world which primarily originates through pathogenic microorganisms rather than immunological or genetical disorders. This review highlights infective mechanisms of non-inflammatory secretory diarrhea causing pathogens, known therapeutics and their efficacy against them. These non-inflammatory diarrheal pathogens breach cell barriers, induce inflammation, disrupt fluid secretion across the epithelium by alteration in ion transport by faulting cystic fibrosis transmembrane conductance regulator (CFTR), calcium activated chloride channels and ion exchanger functions. Currently, a variety of prevention strategies have been used to treat these symptoms like use of antibacterial drugs, vaccines, fluid and nutritional therapy, probiotics and prebiotics as adjuncts. In progression of the need for a therapy having quick physiological effects, withdrawing the symptoms with a wide and safe therapeutic index, newer antisecretory agents like potent inhibitors, agonists and herbal remedies are some of the interventions which have come into light through greater understanding of the mechanisms and molecular targets involved in intestinal fluid secretion. Although these therapies have their own pros and cons inside the host, the quest for new antisecretory agents has been a successful elucidation to reduce burden of diarrheal disease.

PMID: 27473379 [PubMed - as supplied by publisher]

Pf4 bacteriophage produced by Pseudomonas aeruginosa inhibits Aspergillus fumigatus metabolism via iron sequestration.

Microbiology. 2016 Jul 29;

Authors: Penner JC, Ferreira JA, Secor PR, Sweere JM, Birukova M, Joubert LM, Haagensen JA, Garcia O, Malkovskiy AV, Kaber G, Nazik H, Manasherob R, Spormann AM, Clemons KV, Stevens DA, Bollyky PL

Abstract
Pseudomonas aeruginosa (Pa) and Aspergillus fumigatus (Af) are major human pathogens known to interact in a variety of disease settings, including airway infections in cystic fibrosis (CF). We recently reported that clinical isolates of Pa inhibit the formation and growth of Af biofilms. Here we report that the bacteriophage Pf4, produced by Pa, can inhibit the metabolic activity of Af biofilms. This phage-mediated inhibition was dose-dependent, ablated by phage denaturation, and was more pronounced against preformed Af biofilm rather than biofilm formation. In contrast, planktonic conidial growth was unaffected. Two other phages, Pf1 and fd, did not inhibit Af, nor did supernatant from a Pa strain incapable of producing Pf4. Pf4, but not Pf1, attaches to Af hyphae in an avid and prolonged manner, suggesting that Pf4-mediated inhibition of Af may occur at the biofilm surface. We show Pf4 binds iron, thus denying Af a crucial resource. Consistent with this, the inhibition of Af metabolism by Pf4 could be overcome with supplemental ferric iron, with preformed biofilm more resistant to reversal. To our knowledge, this is the first report of a bacterium producing a phage that inhibits the growth of a fungus and the first description of a phage behaving as an iron chelator in a biological system.

PMID: 27473221 [PubMed - as supplied by publisher]

10 new pubmed citations were retrieved for your search. Click on the search hyperlink below to display the complete search results:

("orphan disease" OR "rare disease" OR "orphan diseases" OR "rare diseases")

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11 new pubmed citations were retrieved for your search. Click on the search hyperlink below to display the complete search results:

Pharmacogenomics[Title/Abstract] AND ("2005/01/01"[PDAT] : "3000"[PDAT])

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20 new pubmed citations were retrieved for your search. Click on the search hyperlink below to display the complete search results:

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Concise Review: Cell Surface N-Linked Glycoproteins as Potential Stem Cell Markers and Drug Targets.

Stem Cells Transl Med. 2016 Jul 28;

Authors: Boheler KR, Gundry RL

Abstract
: Stem cells and their derivatives hold great promise to advance regenerative medicine. Critical to the progression of this field is the identification and utilization of antibody-accessible cell-surface proteins for immunophenotyping and cell sorting-techniques essential for assessment and isolation of defined cell populations with known functional and therapeutic properties. Beyond their utility for cell identification and selection, cell-surface proteins are also major targets for pharmacological intervention. Although comprehensive cell-surface protein maps are highly valuable, they have been difficult to define until recently. In this review, we discuss the application of a contemporary targeted chemoproteomic-based technique for defining the cell-surface proteomes of stem and progenitor cells. In applying this approach to pluripotent stem cells (PSCs), these studies have improved the biological understanding of these cells, led to the enhanced use and development of antibodies suitable for immunophenotyping and sorting, and contributed to the repurposing of existing drugs without the need for high-throughput screening. The utility of this latter approach was first demonstrated with human PSCs (hPSCs) through the identification of small molecules that are selectively toxic to hPSCs and have the potential for eliminating confounding and tumorigenic cells in hPSC-derived progeny destined for research and transplantation. Overall, the cutting-edge technologies reviewed here will accelerate the development of novel cell-surface protein targets for immunophenotyping, new reagents to improve the isolation of therapeutically qualified cells, and pharmacological studies to advance the treatment of intractable diseases amenable to cell-replacement therapies.
SIGNIFICANCE: Chemoproteomic techniques that target the cell surfaceome have begun to improve cell immunophenotyping, advance antibody development and usage for sorting, and identify drug targets that advance pharmacological screening and drug repurposing. The development of these techniques and application to stem cells has the potential to accelerate efforts toward the safe use of pluripotent stem cell-derived progeny in regenerative medicine without complicating tumorigenic potential.

PMID: 27471308 [PubMed - as supplied by publisher]

[LIRAGUTIDE AT A DOSE OF 3.0 MG (SAXENDA): NEW INDICATION FOR THE TREATMENT OF OBESITY].

Rev Med Liege. 2016 May;71(5):256-61

Authors: Scheen AJ

Abstract
Liraglutide is an analogue of Glucagon-Like Peptide-1 (GLP-1) already indicated under the trade name of Victoza for the treatment of type 2 diabetes, at usual doses of 1.2 or 1.8 mg as once daily subcutaneous injection. It is henceforth indicated at a dose of 3.0 mg, also as once daily subcutaneous injection, for the treatment of obesity or overweight with comorbidities under the trade name of Saxenda, in combination with diet and exercise. Besides a specific action on the endocrine pancreas, mainly responsible for the antihyperglycaemic effect, liraglutide helps controlling appetite at the hypothamalic level. A specific programme of controlled trials (especially SCALE studies) demonstrated both efficacy and safety of the 3.0 mg dose of liraglutide in obese or overweight patients with various comorbidities.

PMID: 27337846 [PubMed - indexed for MEDLINE]