Provide information about standardization including compositions, quality control, detailed regimen, and duration of treatment. In summary, the reported beneficial effect from Chinese herbs for H1N1 influenza can not be taken as confirmative conclusion. The following methodological issues should be addressed: methods used to generate allocation sequence and allocation GW786034 concealment; double blinding with the use of adequate placebo; clear descriptions of withdrawal/dropout during the trial and use of intention-to-treat analysis; and reporting trials according to the CONSORT Statement . In the literature searching, we identified several registered trials of Chinese herbs for H1N1 influenza. We hope with the publication of these ongoing trials in the future, new high-qualified evidence will arise to provide clinical evidence for the use of Chinese herbs for the H1N1 influenza. Lysosomal storage diseases are a form of metabolic disorder caused by inherited deficiencies of specific lysosomal enzymes, which lead to the accumulation of nonmetabolized macromolecules. The frequency of LSDs as a group varies among populations from 7 to 25 per 100.000. Niemann Pick disease type C1 is a LSD of autosomal recessive inheritance, caused by mutations in the NPC1 gene that encodes for a large transmembrane protein. In Western Europe, the birth incidence of NPC1 has been estimated to be 1 in 150.000. Cells harbouring mutations in NPC1 gene accumulate lowdensity lipoprotein -derived cholesterol in late endosomes/ lysosomes and exhibit defects in lipid trafficking and storage. Affected patients develop ataxia, motor dysfunction and organomegaly. The neuropathological features are characterized by progressive loss of Purkinje cells in the cerebellum, and neurons in the basal ganglia and brain stem. Also, cytoskeletal changes have been demonstrated in neurons in form of neurofibrillary tangles, consisting of hyperphosphorylated tau protein. The initial clinical manifestations of NPC1 disease vary strongly, being neurological, pulmonary or hepatic in nature. The lifespan of patients varies between a few days until over 60 years of age – the majority of cases, however, die between 10 and 25 years of age. One possibility to alleviate the severity of disease could be blocking the intestinal absorption of cholesterol with Ezetimibe or inhibition of protein hyperphosphorylation. Blocking of cyclin-dependent kinases reveal a strong inhibitory effect on protein phosphorylation, being favourable for neural cell survival and thus improving locomotor defects in NPC1 knock-out mice. However, the inhibition of extracellular signal regulated kinases did not alter neurological indices of NPC1 disease in this mouse model. A very promising approach for the treatment of NPC1 is the substrate reduction therapy with the blood-brain barrier crossing small molecule miglustat, which has been reported to reduce lipid accumulation in NPC12/2 mice, thus leading to delay in onset of clinical signs and increasing lifespan.
To ensure evidence-based clinical practice further rigorous placebo-controlled randomized trials are warranted
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