Information about MPS I–IX and related lysosomal storage disorders for patients, families, caregivers, healthcare professionals, and the global community.
This section helps people who may not know which disease they are looking for.
Symptom Information
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Diagnostic Information
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Treatment Options
Brief introduction explaining that treatment-related resources will be available here.
Enzyme Replacement Therapy (ERT)
Small write up about treatment.
Hematopoietic Stem Cell Transplant (HSCT)
Small write up about treatment.
Gene Therapy
Small write up about treatment.
Symptom Management
Small write up about treatment.
Clinical Trials & Research
Stay informed about emerging therapies, clinical trials, and research developments.
Current Clinical Trials
Links to global trial registries.
Research Updates
Latest developments from academic institutions and industry.
Research Participation
Information for families interested in participating.
Newborn Screening Resources
Brief introduction to newborn screening and its relevance to MPS and LSDs.
Educational Resources
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Screening Information
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Country-Specific Resources
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Downloadable Materials
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Genetic Counseling
Understanding inheritance and family planning can help individuals and families make informed decisions.
What is Genetic Counseling?
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Educational Resources
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