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.

Endomyocardial Fibrosis ; Fibroblasts ; Humans ; Myocardium ; pathology

Objective To evaluate the efficacy and safety of etoricoxib and meloxicam in the treatment of patients with acute gout. Methods A randomized,active comparator study was conducted at outpatients and inpatients in our hospital from January 2009 to July 2010.A total of 84patients aged (63.7± 11.0) years with an acute attack of gout were treated with etoricoxib 120 mg/d (n =48),or meloxicam 15 mg/d (n =36) for 7 d.The patient's assessment of joint pain (0- 4 point Likert scale) at drug treatment for 2-5 d was considered as the primary efficacy end point,4 h after firstly takiug the drug and 2-8 d after treatment as the secondary efficacy end point.The starting efficacy was determined until pain relieved by patient himself. The safety was assessed by adverse experiences and indexes including leucocyte, platelet,crcatinine, uric acid,alanine transaminase (ALT),aspartate transaminase (AST) and mean artery pressure(MAP). Results In 84 patients,3cases (8.3％) in meloxicam treatment and 15 cases (31.2％) in etoricoxib treatment (among which 13 cases finished treatment) discontinued therapy.The improvement scores of joint pain were (-0.41 ±0.35 vs.-0.19±0.30,P=0.005) at4 h after firstly taking the drug,(-1.66±0.58 vs. 1.38±0.44,P=0.018)at drug treatment for 2 -5 d,( - 1.83 ± 0.60 vs.- 1.85 ± 0.53,P=0.9) at 2 8 d after treatment,and (-2.64±0.45 vs. - 2.38±0.37,P=0.000) post-treatment higher than pre treatment.The starting time of pain relieving were (4.0 ± 4.6) h in etoricoxib treatment and (12.1±5.7) h in meloxicam treatment. The levels of leucocyte were decreased after treatment as compared with before treatment in both two drug treatments(P＜0.05),while no differences were found in platelet.creatinine,uric acid,ALT and AST.MAP after etoricoxib treatment was increased compared with pretreatment ( P ＜ 0.05 ). Drug related adverse experiences appeared in 15 cases (31.2 ％ ) in etoricoxib treatment and 12 cases(33.3 ％ ) in meloxicam treatment(P=1.000).The ratio of gastrointestinal tract-related adverse effects in meloxicam treatment was higher than in etoricoxib (22.2％ vs.6.2％,P＜ 0.05),while adverse effects on cardiovascular in etoricoxib treatment were comparable to that of meloxicam (16.7 ％ and 11.1 ％,P＞0.05). Conclusions Etoricoxib at a dose of 120 mg once daily may be more effective than meloxicam for acute gout in aspects of safety and tolerance.

Objective To study the relationship between urinary laminin(LN) and diabetic nephropathy.Methods Using the method for concentrating urinary laminin based on precipitation with PEG-4 000 and γ-globulin.The concentration of LN in the urine of 83 diabetics was measured by RIA.The concentration of micro-albumin and Ccr in the urine and the levels of plasma glucose and HbA1c were also measured.Results The choice of 150g/L of PEG-4 000 and 0.5 g/L of γ-globulin was the best method for concentrating urinary LN.The urinary LN in all groups was significantly higher than that in control group.Conclusion Urinary LN is an indicator in the diagnosis of the early diabetic nephropathy.

Objective To analyze and evaluate the effect addition of albumin to the extracorporeal circulation (CPB) in patients undergoing valve replacement. Methods 62 patients under 60 years of age, with the blood level of albumin nearly normal, undergoing mitral valve replacement or aortic valve replacement were randomly divided into two groups. In 34 patients albumin was added to the priming fluid of extraporeal circulation, and in 28 patients it was not. The pre-operative and postoperative serum albumin levels, the duration of assisting ventilation, and the amount of albumin needed between the time of operation to 7 am of the first postoperative day were compared. In both groups the primary priming fluid consisted of balanced electrolyte solution, hydroxyethyl starch, 5% sodium bicarbonate, and 25% mannitol. Results All the indexes, including the preoperative level of albumin, the amount of albumin needed from operation to 7 am of the first postoperative day, and the albumin level at 7 am of the first postoperative day showed no notable differences. Conclusion For patients with no hypoalbuminemia, mitral valve replacement or aortic valve replacement is safe to withhold the addition of albumin to the priming fluid for CPB.

Objective To investigate the expression of nuclear factor-?B(NF-?B) around the hematoma by the intervension of APS in rat, to study mechanisms of APS protecting brain and its effect on inflammation after intracerebral hemorrhage. Methods ICH models was induced in rats by injection ofⅦtype collagenase into the right globas pallidus. APS was used as the intervension drug. Immunohistochemisty was used to investigate the dynamic changes of the expression of NF-?B in the tissue surrounding hematoma in ICH rats. Ultrastructures of the cells around the hematoma were observed with transmission electron microscope. Results Compared with model group, the scores of neurological behavior in treatment group rats were decreased significantly at 3 day and 5 day (P

Objective To investigate the source of CD147 expressing cells in joint and its corelation with rheumatoid arthritis (RA),and to study the expression of CD147 on different cells of peripheral blood and joint fluid of RA patients and normal health controls.Methods The expression rate and intensity of CD147 on lymphocyte (CD3 +T),monocyte (CD14 + Mo) and neutrophils (N) of 10 active RA patients before and after treatment and 10 normal health controls′ peripheral blood and synovial fluid were studied by bicolor immunofluorescence flow cytometry.Results ① Compared with normal control,the expression intensity of CD147 on CD3 + T and CD14 + Mo were higher (P