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61.
J Volden E Duku C Shepherd BA S Georgiades T Bennett B Di Rezze P Szatmari S Bryson E Fombonne P Mirenda W Roberts IM Smith T Vaillancourt C Waddell L Zwaigenbaum M Elsabbagh Pathways in ASD Study Team 《Paediatrics & child health》2015,20(8):e43-e47
OBJECTIVE:
To describe services received by preschool children diagnosed with autism spectrum disorder (ASD) during the five-year period following their diagnosis.METHOD:
An inception cohort of preschoolers diagnosed with ASD from Halifax (Nova Scotia), Montreal (Quebec), Hamilton (Ontario), Edmonton (Alberta) and Vancouver (British Columbia) were invited to participate. Parents/caregivers (n=414) described the services provided to their children at four time points: baseline (T1; within four months of diagnosis; mean age three years); six months later (T2); 12 months later (T3); and at school entry (T4). Data were first coded into 11 service types and subsequently combined into four broader categories (no services, behavioural, developmental and general) for analysis.RESULTS:
More than 80% of children at T1, and almost 95% at T4 received some type of service, with a significant number receiving >1 type of service at each assessment point. At T1, the most common service was developmental (eg, speech-language therapy). Subsequently, the most common services were a combination of behavioural and developmental (eg, intensive therapy based on applied behaviour analysis and speech-language therapy). Service provision varied across provinces and over time.DISCUSSION:
Although most preschool children with ASD residing in urban centres were able to access specialized services shortly after diagnosis, marked variation in services across provinces remains a concern. 相似文献62.
A technique for specific removal of factor IX alloantibodies from human plasma: partial characterization of the alloantibodies 总被引:2,自引:0,他引:2
A method for specific removal of large amounts of factor IX:C alloantibodies by a resin to which highly purified factor IX was linked (factor IX CH-Sepharose) is described. Factor IX was isolated from human plasma by a three-step procedure, including barium citrate adsorption and elution, DEAE-Sepharose CL-6B chromatography, and dextran sulfate agarose chromatography. Approximately 100 mg factor IX was obtained from 60 liters of plasma. The preparation was about 95% pure as judged by SDS-PAA gel electrophoresis. Its specific coagulant activity was 160 U/mg (IX) and its factor IX clotting antigen (IX:Ag) 500-600 U/mg. Essentially quantitative coupling of the factor IX preparation to activated CH-Sepharose 4B was obtained (4 mg factor IX/ml gel; 2300-3000 U/IX:Ag/ml). This resin bound 1500-2000 U factor IX inhibitor/ml gel and could be re-used at least 5 times without any loss in binding capacity. The binding capacity was dependent on the flow rate. No signs of activation of the coagulation, fibrinolytic, or complement system were observed in vitro. Using this factor IX resin, factor IX alloantibodies were isolated and found to consist of two portions, one minor bound to the resin only in the presence of Ca2+ and another major portion Ca2+ independent. The specific inhibitory activity/milligram IgG of the Ca2+-dependent alloantibodies was about 5 times higher in the presence of Ca2+. It is concluded that 25 ml of the factor IX resin described can remove about 40,000 factor IX inhibitor units (comparable to 120,000 Bethesda U) in one run, provided the flow rate does not exceed 20 ml/hr. By using such a technique for removal of antibodies it seems feasible to convert hemophilia-B patients complicated with inhibitors against factor IX into ordinary hemophilia- B patients for treatment at an emergency or in association with major surgery. 相似文献
63.
Tobias Welte R. Phillip Dellinger Henning Ebelt Miguel Ferrer Steven M. Opal Mervyn Singer Jean-Louis Vincent Karl Werdan Ignacio Martin-Loeches Jordi Almirall Antonio Artigas Jose Ignacio Ayestarán Sebastian Nuding Ricard Ferrer Gonzalo Sirgo Rodríguez Manu Shankar-Hari Francisco Álvarez-Lerma Reimer Riessen Josep-Maria Sirvent Stefan Kluge Kai Zacharowski Juan Bonastre Mora Harald Lapp Gabriele Wöbker Ute Achtzehn David Brealey Axel Kempa Miguel Sánchez García Jörg Brederlau Matthias Kochanek Henrik Peer Reschreiter Matthew P. Wise Bernd H. Belohradsky Iris Bobenhausen Benjamin Dälken Patrick Dubovy Patrick Langohr Monika Mayer Jörg Schüttrumpf Andrea Wartenberg-Demand Ulrike Wippermann Daniele Wolf Antoni Torres 《Intensive care medicine》2018,44(4):438-448
Purpose
The CIGMA study investigated a novel human polyclonal antibody preparation (trimodulin) containing?~?23% immunoglobulin (Ig) M,?~?21% IgA, and?~?56% IgG as add-on therapy for patients with severe community-acquired pneumonia (sCAP).Methods
In this double-blind, phase II study (NCT01420744), 160 patients with sCAP requiring invasive mechanical ventilation were randomized (1:1) to trimodulin (42 mg IgM/kg/day) or placebo for five consecutive days. Primary endpoint was ventilator-free days (VFDs). Secondary endpoints included 28-day all-cause and pneumonia-related mortality. Safety and tolerability were monitored. Exploratory post hoc analyses were performed in subsets stratified by baseline C-reactive protein (CRP;?≥?70 mg/L) and/or IgM (≤?0.8 g/L).Results
Overall, there was no statistically significant difference in VFDs between trimodulin (mean 11.0, median 11 [n?=?81]) and placebo (mean 9.6; median 8 [n?=?79]; p?=?0.173). Twenty-eight-day all-cause mortality was 22.2% vs. 27.8%, respectively (p?=?0.465). Time to discharge from intensive care unit and mean duration of hospitalization were comparable between groups. Adverse-event incidences were comparable. Post hoc subset analyses, which included the majority of patients (58–78%), showed significant reductions in all-cause mortality (trimodulin vs. placebo) in patients with high CRP, low IgM, and high CRP/low IgM at baseline.Conclusions
No significant differences were found in VFDs and mortality between trimodulin and placebo groups. Post hoc analyses supported improved outcome regarding mortality with trimodulin in subsets of patients with elevated CRP, reduced IgM, or both. These findings warrant further investigation.Trial registration: NCT01420744.64.
Summary. Patients with moderate and severe haemophilia are evaluated on a regular basis at their haemophilia centres but patients with mild haemophilia are seen less often because of fewer problems related to their disease. The needs of patients with milder forms of haemophilia, however, are often underestimated, both by the patient and staff at healthcare facilities. This study evaluated the knowledge of disease and adherence to treatment among patients with severe, moderate and mild haemophilia. This was a prospective multicentre study performed in Haemophilia Centres in Scandinavia. A total of 413 (67%) of 612 patients aged >25 years with mild, moderate and severe haemophilia completed a self‐administered questionnaire. The mean age of the respondents was 49.7 years (range 25–87 years). Of the 413 respondents, 150 had a mild, 86 had a moderate and 177 had a severe form of haemophilia. A total of 22 (5%) patients did not know the severity of their disease, and 230 (56%) patients knew the effect of factor concentrate in the blood. Of the 413 respondents, 53 (13%) of the cohort never treated a haemorrhage. Patients with mild haemophilia, P ≤ 0.001, were the least likely to treat a haemorrhage. The relative number of patients who were afraid of virus transmission by factor concentrate was about similar in the three groups, 27% of those with severe haemophilia, 26% with moderate and 24% with mild haemophilia. This study shows that the amount of knowledge among haemophilia patients about their disease and treatment is somewhat limited, and demonstrates the importance of continually providing information about haemophilia and treatment, especially to patients with a mild form of the disease. 相似文献
65.
IM Coronel‐Pérez EM Rodríguez‐Rey FM Camacho‐Martínez 《Journal of the European Academy of Dermatology and Venereology》2010,24(4):481-485
Objectives The aim of this study was to test the efficacy of latanoprost in eyelash alopecia areata (AA). Design This study is a 2‐year prospective, non‐blinded, non‐randomized, bilateral eyelash alopecia controlled study. Setting The setting of this study was Trichology Unit, Virgen Macarena University Hospital, Seville, Spain. Patients We conducted a survey of 54 subjects with AA universalis treated with the protocol of the Trichology Unit of our Department. Control group comprised 10 subjects who received injections of 0.5 mg/cm2 of triamcinolone acetonide (TAC) in their eyebrows and 1 mg/cm2 of TAC injections in affected scalp. The treatment group included 44 subjects who received the same treatment as the control group in scalp and eyebrows but they also applied a drop of latanoprost 0.005% (50 μg/mL) ophthalmic solution in their eyelid margins every night. Subjects were reviewed every 3 months for 2 years. Results Forty subjects finished the study and four subjects were lost to follow‐up. In the treatment arm of this study, the course was well tolerated and uncomplicated. Both investigators and patients evaluated the regrowth. The results we obtained were: complete regrowth in 17.5%, moderate regrowth in 27.5%, slight regrowth in 30% and without response in 25%. Moderate and total regrowth constituted a cosmetically acceptable response. The therapy was continuous and the response remained without any side effects. No patients had cosmetically acceptable eyelash regrowth in the control group. Conclusions Latanoprost may be an effective drug in the treatment of eyelash AA because it induces acceptable responses (total and moderate) in 45% of the patients. A formal, blinded prospective unilateral controlled study will permit further understanding about this promising therapeutic agent for eyelash AA. 相似文献
66.
E Fabiani C Catassi A Villari P Gismondi R Pierdomenico IM Rätsch GV Coppa PL Giorgi 《Acta paediatrica (Oslo, Norway : 1992)》1996,85(S412):65-67
In 1992–94 we screened 6315 students for coeliac disease (CD) by testing antigliadin antibodies (AGA) as the first-level investigation. We found 28 biopsy-proven coeliac patients who were invited to start the gluten-free diet (GFD). The aim of this study was a clinical and laboratory follow-up in these screening–detected coeliac adolescents. Patients were 17 females and 11 males with a mean age at diagnosis of 12.8 ± 1 years (range 11–14). Mean follow-up duration time was 23 ± 7 months (range 9–37). Twenty-three of the 28 screening-detected coeliac patients came to the control visit, 3 refused the follow-up and 2 subjects were not found. Twelve patients (52.2%) stated that they never ate any gluten-containing food, while 11 of them (47.8%) reported occasional transgressions to the diet. GFD acceptance was reported as good ( n = 6), moderate ( n = 11) or low ( n = 6). After starting the GFD, signs of improvement were seen in most patients, such as weight gain, increased height velocity and increased feeling of well-being. AGA (both IgG and IgA classes) and antiendomysium antibodies (AEA) were normal in 19 subjects, 2 cases had IgG-AG A and AEA positivity, 1 patient showed abnormal AGA and AEA levels, while isolated IgA-AGA positivity persisted in 1 case. This study shows that even silent CD cases can clinically benefit from the GFD. The consequences of occasional transgressions to the GFD remain unclear. 相似文献
67.
Fifteen patients with infantile bone and joint infections were studied immunologically and clinically, 3 at the time of illness and 12 later. Abnormality of immunoglobulins, or complement, or phagocytes was found in 9 patients; 6 were within normal limits for the tests undertaken. Immunodeficiency is probably responsible for the subdued clinical signs of infection and for delayed diagnosis in some patients. It was also related to the extent of femoral head damage in infective arthritis of the hip and to the incidence of wound infection in late elective surgery. 相似文献
68.
IM Balfour-Lynn I Martin BF Whitehead PG Rees MJ Elliott MR de Leval 《Archives of disease in childhood》1997,76(1):38-40
The outcome of patients with cystic fibrosis aged under 10 years referred for heart-lung transplantation assessment (n = 58) was determined and compared with older children (n = 109). Similar proportions were placed on to the active waiting list (64% v 71%) and received transplants (35% v 31%). Three year post-transplantation survival figures were also similar (41% v 46%), as were the figures for overall survival for those placed on to the active list (27% v 29%). Paediatricians should not be deterred from referring younger patients for transplantation. 相似文献
69.
C Catassi IM Rätsch E Fabiani S Ricci F Bordicchia R Pierdomenico PL Giorgi 《Acta paediatrica (Oslo, Norway : 1992)》1995,84(6):672-676
Many cases of coeliae disease are currently undiagnosed. We carried out a pilot study on screening for coeliae disease in a school population. The screening protocol consisted of three parts: (1) IgG and IgA antigliadin antibody (AGA) assay; (2) antiendomysium antibody and total serum IgA determinations; (3) jejunal biopsy. A total of 5280 students aged 11-15 years (71.7% of the eligible population) underwent the first evaluation; 113 subjects performed the second tests and 35 of these needed the third investigation. Coeliae disease was diagnosed in 23 cases, most of which were atypical or silent forms. The prevalence of undiagnosed coeliae disease was 4.36 per 1000 screened subjects (95% CI 2.58-6.14) and 5.03 per 1000 (95% CI 3.41-6.65) in the general population. The ratio of known to undiagnosed cases was 1 to 6.4. This high prevalence of undiagnosed coeliae disease raises a number of problems that require further evaluation. 相似文献
70.
Close mapping of the focal non-epidermolytic palmoplantar keratoderma (PPK) locus associated with oesophageal cancer (TOC) 总被引:1,自引:1,他引:1
Kelsell DP; Risk JM; Leigh IM; Stevens HP; Ellis A; Hennies HC; Reis A; Weissenbach J; Bishop DT; Spurr NK; Field JK 《Human molecular genetics》1996,5(6):857-860
Focal non-epidermolytic palmoplantar keratoderma (PPK or palmoplantar
ectodermal dysplasia type III) is associated with oesophageal cancer in
three families: two large pedigrees located in Liverpool, UK and in the
midwestern American states and one smaller family from Germany. In these
families, the PPK is inherited as autosomal dominant and has a late onset,
usually manifesting between 7 and 8 years of age. The disease is
characterised by thickening of the pressure areas of the soles, but is not
restricted to the feet and also presents with oral leukokeratosis and
follicular hyperkeratosis. The disease locus [previously termed the
"tylosis oesophageal cancer gene' (TOC) locus] has been mapped to
17q23-qter by linkage analysis. This region is located telomeric to the
keratin 16 gene, in which mutations have been identified in focal PPK
families who show no increased cancer risk. We describe the close mapping
of this locus to the interval between AFMb054zf9 and D17S1603 using
haplotype analysis of additional Genethon markers in the region and show
that although the American family is unlikely to be related to either of
the other two, the UK and German pedigrees may share a common descent. This
work provides a basis for positional cloning and candidate gene analysis in
order to identify a gene that may be involved in familial oesophageal
cancer.
相似文献