Parental perception of treatment options for mucopolysaccharidosis: a survey to bridge the gap for personalized medicine
Abstract Background Mucopolysaccharidosis (MPS) are a group of lysosomal storage diseases with substantial unmet medical needs—for both patients and caregivers. Approved therapies are limited, and the perception of investigative ones remains enigmatic. Method Using an innovative survey concept based...
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| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-01-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-025-03549-y |
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