Wolf B. The neurology of biotinidase deficiency. Mol Genet Metab. 2011;104(1-2):27-34. Wolf B. Clinical issues and frequent questions about biotinidase deficiency. Mol Genet Metab. 2010;100(1):6-13. Zempleni J, Hassan YI, Wijeratne SS. Biotin and biotinidase defi...
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over availab...
01 定义 Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant ...
Herein, for the first time, we reported the clinical features, BTD gene mutations and their functional studies of eight symptomatic children with BTD deficiency from southern China. Fatigue, hypotonia, proximal muscular weakness, hearing deficits, rash and respiratory problems are common clinical ...
BTD Acronyms BTD A gene on chromosome 3p25 that encodes biotinidase, which recycles biotin by cleaving biocytin (biotin-lysine), a normal product of carboxylase degradation. Segen's Medical Dictionary. © 2012 Farlex, Inc. All rights reserved. ...
Master protocol to assess the safety and antitumor activity of genetically engineered T cells in NY-ESO-1 and/or LAGE-1a positive solid tumors (IGNYTE-ESO). ClinicalTrials.gov. Updated October 15, 2024. Accessed January 13, 2025. https://clinicaltrials.gov/study/NCT03967223?tab=table ...
IBI343注册性III期试验|来源:clinicaltrials 胃癌和胰腺癌等肿瘤的靶向治疗手段较匮乏,存在巨大的未满足的临床需求。CLDN18.2高表达于胃癌和胰腺癌中,靶向CLDN18.2的药物是潜在的新型抗肿瘤治疗方案。 目前为止,全球20多家药企布局了claudin 18.2靶向药物的临床开发,大部分在研项目属于单靶点抗体药物,此外还包括claudin...
research and development progresses in China, BTD standards may rise in the future as well. Ultimately, the clinical value of the product will always be the primary consideration. Only products with real breakthrough clinical efficacy will qualify to compete with other breakthrough therapeutic drugs....
9 RegisterLog in Sign up with one click: Facebook Twitter Google Share on Facebook BTD Medical Category filter: AcronymDefinition BTDBefore the Dawn BTDBlood Type Diet BTDBlack Tie Dinner(various organizations) BTDBlue Tooth Dongle BTDBeneficiary Telephone Number Data ...
However, the clinical data which were leveraged in this research is retrospective and the analysis was conducted in limited sample size, statistical conclusion should be interpreted with caution, and confirmation through prospective large cohort studies are warranted to validate these preliminary insights....