Author:
Laney Dawn A.,Gupta Divya,Wechsler Stephanie B.
Publisher
Springer Science and Business Media LLC
Reference99 articles.
1. Meikle PJ, Hopwood JJ, Clague AE, Carey WF. Prevalence of lysosomal storage disorders. JAMA. 1999;281(3):249–54.
2. •• Platt FM. Emptying the stores: lysosomal diseases and therapeutic strategies. Nature Reviews Drug Discovery. 2017. Comprehensive review article focused on disease pathology in LDs, therapeutic targets in LDs, and therapies available or in development in LDs.
3. Vairo FP, Boczek NJ, Cousin MA, Kaiwar C, Blackburn PR, Conboy E, et al. The prevalence of diseases caused by lysosome-related genes in a cohort of undiagnosed patients. Mol Genet Metab Rep. 2017;13:46–51.
4. Orphanet. Orphanet rare diseases collection. Prevalence distribution of rare diseases: bibliographic data. 2016.
http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf
. Accessed 6 Jan 2018.
5. Staretz-Chacham O, Lang TC, LaMarca ME, Krasnewich D, Sidransky E. Lysosomal storage disorders in the newborn. Pediatrics. 2009;123(4):1191–207.
Cited by
3 articles.
订阅此论文施引文献
订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献