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Nonalcoholic steatohepatitis (NASH) can cause end-stage liver diseases such as liver cirrhosis and liver cancer, and therefore, it is urgent to treat NASH, reverse hepatic steatosis, and delay the onset of end-stage liver diseases. NASH has a complex pathogenesis and there are currently no effective drugs for treatment. At present, new drugs still have huge market potential and are the research hotspots of various pharmaceutical companies in China and globally. This article mainly reviews and summarizes the clinical research status, drug types, mechanism of action, and future market prospects of the new drugs for NASH in existing phase Ⅲ studies.
ABSTRACT
Neurogenetic diseases are a group of hereditary diseases that predominantly affect the nervous system, including the central nervous system, peripheral nerves, and muscles. The clinical manifestations are complex, and the treatments are rather difficult. In recent years, with the in-depth study of the etiology and pathogenesis of neurogenetic diseases, gene therapeutic approaches developed for mutant genes, such as antisense oligonucleotides, RNA interference, adeno-associated virus-mediated gene transduction, small molecule compounds with gene modification effects, and clustered regularly interspaced short palindromic repeats/Cas9 gene editing strategies are gradually transformed into clinical applications. The drugs with potential therapeutic effects are developed in according to the promising targets within the pathogenic mechanism of diseases. These emerging therapeutic approaches provide innovative prospects for patients with neurogenetic diseases.
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Objective@#To evaluate the efficacy and safety of a domestic human plasma derived coagulation Factor Ⅸ concentrate (pd-FⅨ) in patients with hemophilia B.@*Methods@#The study was a multicenter, open-label and single-arm study. The efficacy of pd-F Ⅸ was evaluated by objective performance criteria. The doses of pd-FⅨ were calculated according to the bleeding symptom and disease severity. The infusion efficiency of pd-FⅨ and improvement of bleeding symptoms were measured at 30 minutes and (24±4) h after the first infusion, respectively. Adverse events were recorded. Viral infection and FⅨ inhibitor were detected 90 d after the first infusion.@*Results@#All 36 subjects with hemophilia B were enrolled in the study. The median age of these patients was 31 years old and the median injection doses were 4 (1-17) times. The hemostatic effect of 27/36 (75.00%) and 9/36 (25.00%) acute bleeding events were rated as "excellent" and "better" , respectively. The recovery rate was 111.92% (65.55%-194.28%) at 30 minutes after infusion of FⅨ. There was no adverse event related to FⅨ. No reactivation of HBV, HCV or HIV and FⅨ inhibitor was detected at 90-104 d after the first FⅨ infusion.@*Conclusion@#This domestically made human plasma derived FⅨ concentrate is safe and effective in the treatment of acute bleeding in patients with hemophilia B.@*Clinical trial registration@#China food and Durg Administration, 2016L08027.
ABSTRACT
Objective To evaluate the efficacy and safety of domestic human recombinant FSH (rhFSH) in women with anovulation of WHO groupⅡ. Methods A randomized, blind, parallel-controlled, non-inferiority and multicenter study was performed. A total of 534 admitted to 13 hospitals from May 2008 to August 2009. There were 531 women with ovulatory disorder was included in the statistical analysis, were randomly divided into test group (domestic rhFSH, n=352) and control group (imported rhFSH, n=179). Percentage of cycle with mature follicle, ovulation rate, clinical pregnancy rate, multiple pregnancy rate, ovarian hyperstimulation syndrome (OHSS) and adverse events were observed. Results No statistical significant differences (P>0.05) were observed between the two groups in terms of the efficiency on mature follicle [91.8%(323/352) versus 88.8%(159/179)], ovulation rate [91.3%(295/323) verus 90.6%(144/159)], clinical pregnancy rate [19.2%(62/323) verus 18.2%(29/159)], the number of the follicles0.05), and no other adverse events were observed in test group during treatment. Conclusion Ovarian stimulation with domestic rhFSH is effective, safe and economical in women with anovulation of WHO groupⅡ.