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https://www.readbyqxmd.com/read/28644430/the-clinical-landscape-for-sma-in-a-new-therapeutic-era
#1
REVIEW
K Talbot, E F Tizzano
Despite significant advances in basic research, the treatment of degenerative diseases of the nervous system remains one of the greatest challenges for translational medicine. The childhood onset motor neuron disorder spinal muscular atrophy (SMA) has been viewed as one of the more tractable targets for molecular therapy, due to a detailed understanding of the molecular genetic basis of the disease. In SMA, inactivating mutations in the SMN1 gene can be partially compensated for by limited expression of SMN protein from a variable number of copies of the SMN2 gene, which provides both a molecular explanation for phenotypic severity and a target for therapy...
June 23, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28642642/access-to-and-payment-for-office-based-buprenorphine-treatment-in-ohio
#2
Theodore V Parran, Joseph Z Muller, Elina Chernyak, Chris Adelman, Christina M Delos Reyes, Douglas Rowland, Mykola Kolganov
IMPORTANCE: Office-based opiate agonist therapy has dramatically expanded access to medication-assisted treatment over the past decade but has also led to increased buprenorphine diversion. OBJECTIVE: Our study sought to characterize physicians who participate in office-based therapy (OBT) to assess patient access to OBT in Ohio 10 years after its introduction. DESIGN/SETTING/PARTICIPANTS: Cross-sectional telephone survey of Drug Addiction Treatment Act-waivered physicians in Ohio listed by the Center for Substance Abuse Treatment (CSAT)...
2017: Substance Abuse: Research and Treatment
https://www.readbyqxmd.com/read/28641353/-negative-pressure-wound-therapy-randomised-controlled-trials-from-2000-to-2015
#3
Frank Peinemann
Background Negative pressure wound therapy is believed to promote wound healing. However, the results from randomised controlled trials (RCTs) are inconsistent. Systematic reviews indicate unclear evidence, due in part to low-quality RCTs, calling for more RCTs of higher quality. Methods This study aimed to ascertain the number of RCTs as well as systematic reviews on RCTs published during the time period from 2000 to 2015. Various search strategies were used to identify studies in PubMed, Cochrane Library, Google, references lists of retrieved articles, and institutions that issue evidence reports...
June 2017: Zentralblatt Für Chirurgie
https://www.readbyqxmd.com/read/28640730/treating-patients-with-venous-leg-ulcers-in-the-acute-setting-part-1
#4
Irene Anderson
Leg ulcers present with a variety of aetiologies, sometimes in combination. The most common aetiology is venous, with treament involving compression, elevation and exercise; the most common treatment setting is the community. However, people with leg ulcers do sometimes require admission to hospital for conditions and situations which may, or may not, be ulcer-related. There is a lack of contemporary evidence on the experience of inpatients and insufficient analysis of the impact on healing and complications to the lower limb when patients with leg ulcers and compression therapy are admitted to hospital...
June 22, 2017: British Journal of Nursing: BJN
https://www.readbyqxmd.com/read/28639990/recent-mobile-health-interventions-to-support-medication-adherence-among-hiv-positive-msm
#5
Kathryn E Muessig, Sara LeGrand, Keith J Horvath, José A Bauermeister, Lisa B Hightow-Weidman
PURPOSE OF REVIEW: We describe recent mobile health (mHealth) interventions supporting antiretroviral therapy (ART) medication adherence among HIV-positive MSM. RECENT FINDINGS: Keyword searches (1 January 2016-13 May 2017) identified 721 citations. Seven publications reporting on six studies met inclusion criteria. Five studies focused on MSM. Interventions primarily employed text messaging (n = 4), whereas two focused on smartphone apps and one on social media...
July 20, 2017: Current Opinion in HIV and AIDS
https://www.readbyqxmd.com/read/28639459/approach-to-the-management-of-patients-with-hidradenitis-suppurativa-a-consensus-document
#6
Afsaneh Alavi, Charles Lynde, Raed Alhusayen, Marc Bourcier, Isabelle Delorme, Ralph George, Melinda Gooderham, Wayne Gulliver, Sunil Kalia, Danielle Marcoux, Yves Poulin
BACKGROUND: Hidradenitis suppurativa (HS) is a painful, debilitating, and poorly understood condition, which is suboptimally diagnosed, managed, and treated. Evidence supporting various treatment modalities is sparse. OBJECTIVES: To incorporate scientific evidence and expert opinions to develop useful guidance for the evaluation and management of patients with HS. METHODS: An expert panel of Canadian dermatologists and surgeons developed statements and recommendations based on available evidence and clinical experience...
June 1, 2017: Journal of Cutaneous Medicine and Surgery
https://www.readbyqxmd.com/read/28637073/-complementary-and-alternative-medicine-in-oncology-what%C3%A2-makes-sense
#7
Axel Eustachi, Dieter Melchart
The basis of any complementary therapy should be the recommendation of a healthy lifestyle (diet, nutrition, exercise, stress-reduction), and appropriate self-help techniques (e. g. thermotherapy, herbal OTC-products) aimed at releasing the patients' most debilitating symptoms. For therapy-resistant or strong symptoms, more invasive interventions or medical treatments with an acceptable evidence base can be used (e. g. acupuncture for cancer pain, herbal prescriptions). The effects of any complementary intervention should be controlled for a period of six weeks after its administration...
June 2017: Deutsche Medizinische Wochenschrift
https://www.readbyqxmd.com/read/28634906/delivery-of-brief-cognitive-behavioral-therapy-for-medically-ill-patients-in-primary-care-a-pragmatic-randomized-clinical-trial
#8
Jeffrey A Cully, Melinda A Stanley, Nancy J Petersen, Natalie E Hundt, Michael R Kauth, Aanand D Naik, Kristen Sorocco, Shubhada Sansgiry, Darrell Zeno, Mark E Kunik
BACKGROUND: Few studies have examined the practical effectiveness and implementation potential of brief psychotherapies that integrate mental and physical health. OBJECTIVE: To determine whether an integrated brief cognitive behavioral therapy (bCBT), delivered by mental health providers in primary care, would improve depression, anxiety and quality of life for medically ill veterans. DESIGN: Pragmatic patient-randomized trial comparing bCBT to enhanced usual care (EUC)...
June 20, 2017: Journal of General Internal Medicine
https://www.readbyqxmd.com/read/28634878/can-music-based-movement-therapy-improve-motor-dysfunction-in-patients-with-parkinson-s-disease-systematic-review-and-meta-analysis
#9
Shuai Zhang, Dong Liu, Dan Ye, Haiyu Li, Feng Chen
This study aimed to quantify whether there is association between music-based movement therapy and motor dysfunction in patients with Parkinson's disease, and, if so, whether music-based movement therapy can be used as first-line non-pharmacological treatment. To conduct a systematic review and meta-analysis of clinical trials that examined the effect of music-based movement therapy on patient-relevant and disease-specific outcomes. Comprehensive literature was searched of PubMed, EMbase, and the Cochrane Library from inception to November 2016...
June 21, 2017: Neurological Sciences
https://www.readbyqxmd.com/read/28633504/a-phase-ii-study-of-palonosetron-aprepitant-dexamethasone-and-olanzapine-for-the-prevention-of-cisplatin-based-chemotherapy-induced-nausea-and-vomiting-in-patients-with-thoracic-malignancy
#10
Kazuhisa Nakashima, Haruyasu Murakami, Kouichi Yokoyama, Shota Omori, Kazushige Wakuda, Akira Ono, Hirotsugu Kenmotsu, Tateaki Naito, Fumie Nishiyama, Mami Kikugawa, Masayo Kaneko, Yumiko Iwamoto, Satomi Koizumi, Keita Mori, Takeshi Isobe, Toshiaki Takahashi
Background: The three-drug combination of a 5-hydroxytryptamine type 3 receptor antagonist, a neurokinin 1 receptor antagonist and dexamethasone is recommended for patients receiving highly emetogenic chemotherapy. However, standard antiemetic therapy is not completely effective in all patients. Methods: We conducted an open-label, single-center, single-arm Phase II study to evaluate the efficacy of olanzapine in combination with standard antiemetic therapy in preventing chemotherapy-induced nausea and vomiting in patients with thoracic malignancy receiving their first cycle of cisplatin-based chemotherapy...
June 17, 2017: Japanese Journal of Clinical Oncology
https://www.readbyqxmd.com/read/28628931/-the-probabilistic-efficiency-frontier-a-value-assessment-of-treatment-options-in-hepatitis-c
#11
Axel C Mühlbacher, Andrew Sadler
Background The German Institute for Quality and Efficiency in Health Care (IQWiG) recommends the concept of the efficiency frontier to assess health care interventions. The efficiency frontier supports regulatory decisions on reimbursement prices for the appropriate allocation of health care resources. Until today this cost-benefit assessment framework has only been applied on the basis of individual patient-relevant endpoints. This contradicts the reality of a multi-dimensional patient benefit. Objective The objective of this study was to illustrate the operationalization of multi-dimensional benefit considering the uncertainty in clinical effects and preference data in order to calculate the efficiency of different treatment options for hepatitis C (HCV)...
June 19, 2017: Das Gesundheitswesen
https://www.readbyqxmd.com/read/28627356/aquatic-therapy-for-children-with-duchenne-muscular-dystrophy-a-pilot-feasibility-randomised-controlled-trial-and-mixed-methods-process-evaluation
#12
Daniel Hind, James Parkin, Victoria Whitworth, Saleema Rex, Tracey Young, Lisa Hampson, Jennie Sheehan, Chin Maguire, Hannah Cantrill, Elaine Scott, Heather Epps, Marion Main, Michelle Geary, Heather McMurchie, Lindsey Pallant, Daniel Woods, Jennifer Freeman, Ellen Lee, Michelle Eagle, Tracey Willis, Francesco Muntoni, Peter Baxter
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare disease that causes the progressive loss of motor abilities such as walking. Standard treatment includes physiotherapy. No trial has evaluated whether or not adding aquatic therapy (AT) to land-based therapy (LBT) exercises helps to keep muscles strong and children independent. OBJECTIVES: To assess the feasibility of recruiting boys with DMD to a randomised trial evaluating AT (primary objective) and to collect data from them; to assess how, and how well, the intervention and trial procedures work...
May 2017: Health Technology Assessment: HTA
https://www.readbyqxmd.com/read/28626219/base-excision-repair-proteins-couple-activation-induced-cytidine-deaminase-and-endonuclease-g-during-replication-stress-induced-mll-destabilization
#13
B Gole, E Mian, M Rall, L Wiesmüller
The breakpoint cluster region of the MLL gene (MLLbcr) is frequently rearranged in therapy-related and infant acute leukaemia, but the destabilizing mechanism is poorly understood. We recently proposed that DNA replication stress results in MLLbcr cleavage via Endonuclease G (EndoG) and represents the common denominator of genotoxic therapy-induced MLL destabilization. Here we performed a siRNA screen for new factors involved in replication stress-induced MLL rearrangements employing an EGFP-based reporter system...
June 19, 2017: Leukemia: Official Journal of the Leukemia Society of America, Leukemia Research Fund, U.K
https://www.readbyqxmd.com/read/28625650/switch-maintenance-chemotherapy-using-s-1-with-or-without-bevacizumab-in-patients-with-advanced-non-small-cell-lung-cancer-a-phase-ii-study
#14
Seiji Niho, Yuichiro Ohe, Hironobu Ohmatsu, Shigeki Umemura, Shingo Matsumoto, Kiyotaka Yoh, Koichi Goto
OBJECTIVES: We conducted this single-institute; prospective, non-randomized parallel two-arm phase II study to evaluate the efficacy and safety of switch maintenance chemotherapy with S-1 after induction therapy with a platinum-based regimen in patients with advanced non-small cell lung cancer (NSCLC). PATIENTS AND METHODS: Patients not showing disease progression after induction platinum-based chemotherapy received S-1 at the dose of 40mg/m(2) twice daily for 14 consecutive days, every three weeks, with or without bevacizumab (Bev) at the dose of 15mg/kg...
June 2017: Lung Cancer: Journal of the International Association for the Study of Lung Cancer
https://www.readbyqxmd.com/read/28620930/prospective-multimodal-imaging-assessment-of-locally-advanced-cervical-cancer-patients-administered-by-chemoradiation-followed-by-radical-surgery-the-price-prospective-imaging-of-cervical-cancer-and-neoadjuvant-treatment-study-the-role-of-ultrasound
#15
A C Testa, G Ferrandina, F Moro, T Pasciuto, M C Moruzzi, I De Blasis, F Mascilini, E Foti, R Autorino, A Collarino, B Gui, G F Zannoni, M A Gambacorta, A L Valentini, V Rufini, G Scambia
OBJECTIVE: Chemoradiation-based neoadjuvant treatment followed by radical surgery is an alternative therapeutic strategy for locally advanced cervical cancer (LACC) -stage ≥ IB2 (except of IIA1)- but ultrasound variables used to predict neoadjuvant partial response are not well defined. The goal of this study was to prospectively analyze the potential role of transvaginal ultrasound (TVUS) in early predicting pathologically assessed residual disease in a large, single-institution series of LACC patients triaged to neoadjuvant treatment followed by radical surgery...
June 16, 2017: Ultrasound in Obstetrics & Gynecology
https://www.readbyqxmd.com/read/28620323/integrated-trauma-focused-cognitive-behavioural-therapy-for-post-traumatic-stress-and-psychotic-symptoms-a-case-series-study-using-imaginal-reprocessing-strategies
#16
Nadine Keen, Elaine C M Hunter, Emmanuelle Peters
Despite high rates of trauma in individuals with psychotic symptoms, post-traumatic stress symptoms are frequently overlooked in clinical practice. There is also reluctance to treat post-traumatic symptoms in case the therapeutic procedure of reprocessing the trauma exacerbates psychotic symptoms. Recent evidence demonstrates that it is safe to use reprocessing strategies in this population. However, most published studies have been based on treating post-traumatic symptoms in isolation from psychotic symptoms...
2017: Frontiers in Psychiatry
https://www.readbyqxmd.com/read/28619061/rituximab-versus-cyclophosphamide-for-the-treatment-of-connective-tissue-disease-associated-interstitial-lung-disease-recital-study-protocol-for-a-randomised-controlled-trial
#17
Peter Saunders, Vicky Tsipouri, Gregory J Keir, Deborah Ashby, Marcus D Flather, Helen Parfrey, Daphne Babalis, Elisabetta A Renzoni, Christopher P Denton, Athol U Wells, Toby M Maher
BACKGROUND: Interstitial lung disease (ILD) frequently complicates systemic autoimmune disorders resulting in considerable morbidity and mortality. The connective tissue diseases (CTDs) most frequently resulting in ILD include: systemic sclerosis, idiopathic inflammatory myositis (including dermatomyositis, polymyositis and anti-synthetase syndrome) and mixed connective tissue disease. Despite the development, over the last two decades, of a range of biological therapies which have resulted in significant improvements in the treatment of the systemic manifestations of CTD, the management of CTD-associated ILD has changed little...
June 15, 2017: Trials
https://www.readbyqxmd.com/read/28619000/astrocytes-pathology-in-als-a-potential-therapeutic-target
#18
Sonja Johann
The mechanisms underlying neurodegeneration in amyotrophic lateral sclerosis (ALS) are multifactorial and include genetic and environmental factors. Nowadays, it is well accepted that neuronal loss is driven by non-cell autonomous toxicity. Non-neuronal cells, such as astrocytes, have been described to significantly contribute to motoneuron cell death and disease progression in cell culture experiments and animal models of ALS. Astrocytes are essential for neuronal survival and function by regulating neurotransmitter and ion homeostasis, immune response, blood flow and glucose uptake, antioxidant defence and growth factor release...
June 15, 2017: Current Pharmaceutical Design
https://www.readbyqxmd.com/read/28618158/a-fitbit-and-facebook-mhealth-intervention-for-promoting-physical-activity-among-adolescent-and-young-adult-childhood-cancer-survivors-a-pilot-study
#19
Jason A Mendoza, K Scott Baker, Megan A Moreno, Kathryn Whitlock, Mark Abbey-Lambertz, Alan Waite, Trina Colburn, Eric J Chow
BACKGROUND: Physical activity (PA) may be important for preventing chronic diseases for adolescent and young adult (AYA) childhood cancer survivors. Randomized controlled trials (RCTs) of PA interventions for AYA survivors are sparse, but necessary to determine effective programs for increasing PA among this population. Thus, we conducted a pilot RCT, testing the feasibility of a mobile health (mHealth) intervention to promote PA among AYA survivors. PROCEDURE: We recruited 14- to 18-year-olds who were ≥1-year post cancer therapy from Seattle Children's Hospital...
June 15, 2017: Pediatric Blood & Cancer
https://www.readbyqxmd.com/read/28618075/surgical-treatment-for-advanced-hepatocellular-carcinoma-with-portal-vein-tumor-thrombus
#20
REVIEW
Kazuhiko Sakamoto, Hiroaki Nagano
The Barcelona Clinic Liver Cancer (BCLC) staging system recommends administration of a tyrosine kinase inhibitor (sorafenib) as standard therapy in advanced hepatocellular carcinoma (HCC) patients with portal vein tumor thrombus (PVTT). Sorafenib has been shown to prolong median overall survival (OS) by about 3 months in advanced HCC patients with PVTT (8.1 vs 4.9 months). However, its clinical effectiveness is still controversial and standard treatment with sorafenib is not established in Japan. Surgical resection is considered as a potentially curative treatment and provides an acceptable outcome for carefully selected patients...
June 15, 2017: Hepatology Research: the Official Journal of the Japan Society of Hepatology
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