A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life
Abstract Background Krabbe disease is a rare neurodegenerative disorder caused by a deficiency in the lysosomal enzyme galactocerebrosidase. Patients with Krabbe disease present with a variable disease course depending on their age of onset. The purpose of this prospective cohort study was to charac...
محفوظ في:
| المؤلفون الرئيسيون: | , , , |
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| التنسيق: | Artigo |
| اللغة: | Inglês |
| منشور في: |
BMC
2018-08-01
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| سلاسل: | Orphanet Journal of Rare Diseases |
| الموضوعات: | |
| الوصول للمادة أونلاين: | http://link.springer.com/article/10.1186/s13023-018-0872-9 |
| الوسوم: |
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