The United States is the first country that has introduced legislation designed to stimulate orphan drug research and development .In this paper , we analyze three major amendments of the Orphan Drug Act ( ODA) since 1983 and its incentive system .We then discuss the success of the ODA in encouraging orphan drug research and development and boosting the biopharmaceutical industry .Following that , we investigate the high price of orphan drugs caused by market exclusivity and drug repositioning .Finally, we draw experience from the ODA to make some suggestions on how orphan drug legislation can be devised in China with the aim of improving the health of rare -dis-ease patients and facilitating the development of Chinese biopharmaceutical industry .

Objective To investigate the relationship between cerebrovascular accident and abnormal electrocardiogram. Methods The ECG abnormalities and clinical data of 314 patients with cerebrovascular accident were analyzed. Results 56.7% patients developed arrhythmia. Among them, the morbility of arrhythmia in patients with cerebral hemorrhage was higher than that of cerebral infarction ( P

OBJECTIVETo investigate the effects of recombinant human growth hormone on serum lipid in aged male patients with chronic heart failure (CHF).

METHODSEighty seven patients with chronic heart failure(> or = 60 years old) were randomly divided into 2 groups: the CHF control group (n = 46) who received regular therapy and the CHF experimental group (n = 41) who received regular therapy and recombinant human growth hormone. The treatment would be continued for 3 months. Another group was normal control group (n = 10). The detection of serum growth hormone (GH), insulin-like growth factor (IGF-1), total cholesterol (TC), triglyceride (TG), low-density lipoprotein cholesterol (LDL-C), high density lipoprotein cholesterol (HDL-C) was carried out before and after treatment in the participants.

RESULTSBefore treatment, the levels of GH and IGF-1 were not significantly different among groups. After treatment, the levels of GH (0.71 +/- 0.34 vs 0.96 +/- 0.48) and IGF-1 (95.64 +/- 21.11 vs 111.64 +/- 23.14)in CHF experimental group were higher than those before the treatment. In CHF control group, the levels of GH(0.81 +/- 0.32 vs 0.79 +/- 0.29) and IGF-1 (97.82 +/- 19.74 vs 99.65 +/- 20.11) had no significant change after the treatment. After treatment, the levels of GH (0.96 +/- 0.48 vs 0.79 +/- 0.29) and IGF-1 (111.64 +/- 23.14 vs 99.65 +/- 20.11) in CHF experimental group were higher compared with that of CHF control group. Before treatment, the serum levels of LDL-C, HDL-C, TC and TG had no significant difference among groups. After treatment,the levels of LDL-C (2.11 +/- 0.82 vs 1.76 +/- 0.51) and TC (3.78 +/- 1.34 vs 3.21 +/- 1.17) in CHF experimental group were lower than those before the treatment. However, the levels of HDL-C (1.10 +/- 0.31 vs 0.99 +/- 0.28)and TG (1. 89 +/- 1.07 vs 1.66 +/- 0.95) had no significant change after the treatment compared with before treatment. In CHF control group, the serum lipid levels had no significant change after the treatment.

CONCLUSIONAs the treatment of rhGH for aged male patients with chronic heart failure, GH influences lipid metabolism, which reduces the level of LDL-C, TC. However GH has no effects on the serum HDL-C and TG level. With the treatment of rhGH for long-term, lipid metabolism should be paid attention,and the treatment for blood lipid reduction should be adjusted in time.

Aged ; Chronic Disease ; Heart Failure ; blood ; therapy ; Human Growth Hormone ; pharmacology ; Humans ; Lipids ; blood ; Male ; Recombinant Proteins ; pharmacology

Objective To investigate the clinical features and therapy of fournier's gangrene.Methods The clinical data of 12 patients with fournier's gangrene who were treated at the Third Affiliated Hospital of Nanjing University of Chinese Medicine from January 2010 to February 2012 were retrospectively analyzed.Eleven patients had perianal abscess and 1 patient had perianal foreign body,most of the patients were presented with perianal pain,fever (＞ 38.5 ℃) and tachycardia.The mean interval between the onset of symptoms and admission to the hospital was 8 days (range,3-20 dyas).Seven patients were complicated with diabetes.Only 2 patients were administered hypoglycemic agents,but the effects were poor.Six patients were complicated with hypertension.One patient had the history of resection of sigmoid colon cancer 2 months before operation.Debridement and drainage were applied to all the patients after examination.Antimicrobial therapy applied to all the patients according to the results of drug sensitivity test.The incisions were washed by hydrogen peroxide solution postoperatively.Redebridement was applied if the disease was progressed.All patients were followed up via out-patient examination and phone call till June 2012.Results Results of clinical features:Perianal tissues necrotized,with the color of black.The scrotum or labia majora of 8 patients were invaded by the necrotic tissues.Results of laboratory test:the white blood cell count was (3.8-27.6) × 109/L,the white blood cell count of 6 patients was above 10 × 109/L,3 was above 20 × 109/L.The ratio of neutrophil granulocytes was 0.61-0.93.Four patients received single debridement,5 received redebridement,and 3 patients received debridement for more than 2 times.Two patients had sapremia and infectious shock,and they received treatment for 3 days and 4 days at the intensive care unit.Results of bacterial culture:6 patients were infected by the escherichia coli,4 by klebsiella pnenmoniae,and 1 by pseudomonas aeruginosa.The mean duration of hospital stay was (25± 14)days (range,1-49 days).All patients were followed up for 4-29 months,1 patient was discharged at postoperative day 2,and the prognosis of the 11 patients was good,no mortality was observed.Conclusions The clinical features of fournier's gangrene include severe pain and rapid spread of infections caused by necrosis of perianal subcutaneous tissues.Diabetes might be the risk factor of fournier's gangrene.Early diagnosis and prompt and aggressive surgical debridement are critical for improving survival.

Objective To systematic review the efficacy of Bifidobacterium preparation in Helicobacter pylori ( HP ) eradication therapy. Methods We systematically reviewed clinical research about the efficacy of probiotics in Helicobacter pylori eradication in multiple data-base( PubMed, EMBASE, Medline, OVID, Web of Science); After screening and assessing the quality of the data, we used RevMan 5.3.5 software and Stata 12.0 software for data analysis, then we used GRADE pro3.6.1 software assessing the quality of results.Results Six studies were included with 1396 patients,there were 690 patients in experimental group and 706 patients in control group.Compared with control group, the eradication rates calculated by per-protocol analysis [RR=1.19,95%CI(1.12,1.26),Z=5.87 (P<0.00001)]and intention-to-treat analysis[RR=1.18,95%CI(1.07,1.31),Z=3.33(P=0.0009)]in experimental group was higher,the number of diarrhea[RR=0.43,95%CI(0.23,0.79),Z=2.73(P=0.006)]/the number of nausea[RR=0.67,95%CI(0.56,0.81),Z=4.19 (P<0.0001)]/the number of taste disorders[RR=0.61,95%CI(0.32,1.13),Z=1.57(P=0.12)]in experimental group was lower.The quality of results:the eradication rates calculated by per-protocol analysis was high quality, intention-to-treat analysis and the number of nausea and the number of diarrhea was moderate quality,the number of taste disorders was low quality.Egger’s test showed there was no evidence of substantial publication bias.Conclusion Bifidobacterium preparations during standard triple HP therapy may improve the eradication rate and reduce adverse reactions.

[Objective]To investigate the compatibility of tissue and cell in the three-dimensional porous bioactive glass/recombine human Bone Morphogenetic Protein-2 (BG /rhBMP2)materialscaffolds and provide a reference for improving the constitution of composite material.[Method]The material of BG and BG/rhBMP2 were cultured in vitro with rabbit osteoblasts respectively. The morphology and adhesion were examined by inverted microscope and scanning electron microscope, and the cell proliferation was detected by MTT assay. The composite materialscaffold were embeded in rabbit's muscle and incisions were observed. The specimens were harvested in 2, 4, 8 and 12 weeks respectively and examined by histology to evaluate the tissue response.[Result]The results showed the cells on BG and BG /rhBMP2 were good adhension and activity, the two materialscaffolds had no apparent inflammatory reaction in vivo. MTT assay showed the number of the cells in each group were significantly increased along with the culture time prolonged. The number of cells in the BG and rhBMP2 group was higher than that of the control group at different intervals, with a significant difference. The number of cells in the BG/rhBMP2 was not evidently higher than that of the BG with a significant difference.[Conclusion]The porous composite materialscaffold of BG/rhBMP2 has good biocompatibility with the hope to wide use in bone tissue engineering.

AIM: To investigate the expression of c-fos in the neonatal rats' brains following hypoxia-ischemia.METHODS: RT-PCR and immunohistochemistry were used to detect the transcription and translation of c-fos gene in the cortex and hippocampus following hypoxia-ischemia.RESULTS: The expression of c-fos mRNA and protein were induced in the cortex and hippocampus at the early stage following hypoxia-ischemia(P

Objective Intramuscular ketamine is often used for premedication in children. Premeditation can also be administered perorally in children. The aim of this study was to evaluate the efficacy of different compounds of ketamine given perorally as premedication in children. Methods Seventy-five ASA Ⅰ- Ⅱ pediatnc patients weighing 10-30 kg undergoing urologic operation were randomly divided into 5 groups of 15 patients each : (1) control group received atropme 0.015 mg ? kg-1 im 30 min before surgery; (2) DA group received intramuscular diazepam 0.2 mg?kg-1 and atropine 0.015 mg?kg-1 30 min before operation; (3) (4) (5) KMA groups received ketamine 3 mg?kg-1 (K3MA) or5mg?kg-1 (K5MA) or 8mg?kg-1 ( K8 MA) + midazolam 0.5 mg?kg-1 + atropine 0.03 mg?kg per os 30 min before operation. SpO2 and heart rate (HR) were monitored and recorded before premedication and at 0, 5, 10, 15, 20, 30 and 40 min after premedication. Peak effect time, duration of operation and emergence time were also recorded. Sedation, anxiolysis and behaviour at separation from parents, during venepuncture and induction were graded and assessed. Results There was no significant difference in duration of operation among the five groups. The peak effect time in the three KMA groups was shorter than that in control and DA group and was shortest in K8MA group. The three KMA groups were significantly better than control and DA group and the K8 MA group was the best in terms of sedation, anxiolysis and analgesia. The incidence of adverse effects like diploplia headache and agitation was higher in K8MA group. Conclusion K5MA group provides satisfactory sedation and analgesia similer to Kg MA group with less side-effects, so is the oral ketamine compound of choice for premedication in children.

Cardiovascular System ; Pharmacogenetics

Endomyocardial Fibrosis ; Fibroblasts ; Humans ; Myocardium ; pathology