Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials
<strong>Background:</strong> Niemann-Pick disease type C (NPC) is a lysosomal storage disease with a heterogeneous neurodegenerative clinical course. Multiple therapies are in clinical trials and inclusion criteria are currently mainly based on age and neurological signs, not taking into...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Journal article |
| Published: |
BioMed Central
2018
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