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线粒体肌病
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Bax, B.E (2017) Drug Development for Rare Diseases: Challenges and Regulatory Initiatives. Archives of Science, 1:2
https://www.omicsonline.org/open-access/drug-development-for-rare-diseases-challenges-and-regulatory-initiatives.pdf
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研究者
Chapman, T.P., Hadley, G., Fratter, C., Cullen, S., Bax, B.E., Bain, M.D., Sapsford, A., Poulton J., and Travis, S.P.L. (2014) Unexplained gastrointestinal symptoms: Think mitochondrial myopathy. Digestive and Liver Disease. 46, 1-8.
https://www.ncbi.nlm.nih.gov/pubmed/23768727
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研究者
Bax, B.E., Bain, M.D., Scarpelli, M., Filosto, M., Tonin P., and Moran, N. F. (2013) Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement. Neurology. 8, 1269-1271.
https://www.ncbi.nlm.nih.gov/pubmed/23966250
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研究者
Levene, M., Coleman, D., Kilpatrick, H., Fairbanks, L., Gangadharan, B., Gasson, C., and Bax, B.E. (2013) Preclinical toxicity evaluation of erythrocyte-encapsulated thymidine phosphorylase in BALB/c mice and Beagle dogs: an enzyme replacement therapy for
https://www.ncbi.nlm.nih.gov/pubmed/22977166
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研究者
Scarpelli, M., Russignan, A., Zombor, M., Bereczki, C., Zappini, F., Buono, R., Bax, B.E., Padovani, A., Tonin P., and Filosto, M (2012) . Poor outcome in a MNGIE Patient with a novel TYMP mutation: the need for early diagnosis. Case Reports in Neurolo
https://www.ncbi.nlm.nih.gov/pubmed/23341816
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研究者
Bourgeaux, V., Lanao, J.M., Bax, B.E., and Godfrin, Y. (2016) Drug-loaded erythrocytes: on the road toward marketing approval. Drug design Development and Therapy. 10, 665-676.
https://www.ncbi.nlm.nih.gov/pubmed/26929599
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研究者
Zespół Leigha - miopatia mitochondrialna
https://chorobyrzadkie.blogspot.com/2012/12/zespo-leigha-miopatia-mitochondrialna.html
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专业健康护理