Get involved: make a difference in Hurler syndrome (MPS I-H)

Why your involvement is so important

Hurler syndrome is extremely rare, which means that every experience, data point and voice matters. When families and professionals participate in registries, share insights and support advocacy, they help shape better care and future treatments for everyone affected.

• Better evidence: more complete data strengthens research and clinical guidance.
• Stronger advocacy: policy makers respond when data and lived experience align.
• Improved services: feedback helps identify gaps and improve care pathways.
• Future therapies: high-quality outcome data is essential for evaluating new treatments, including gene therapy.

How families and carers can help

• Ask about registries: speak with your specialist team about relevant national or international registries.
• Share information over time: attend follow-up visits and complete quality-of-life or experience questionnaires.
• Join a support organisation: connect with MPS or rare disease charities involved in data and advocacy work.
• Tell your story if you wish: personal experiences help explain what the numbers mean.
• Take part in research: consider studies focused on education, mental health, care pathways or daily life.

How healthcare professionals can contribute

• Participate in registries and long-term follow-up programmes.
• Refine local care pathways using guidelines and registry insights.
• Collaborate with patient organisations on clear, family-friendly materials.
• Support advocacy by sharing evidence, summaries or expert perspectives.
• Teach and mentor colleagues to raise awareness of Hurler syndrome.

How researchers can drive progress

• Design projects aligned with priority areas such as CNS involvement and long-term outcomes.
• Use and strengthen registries to improve data quality and relevance.
• Share findings clearly with both scientific and non-scientific audiences.
• Collaborate across centres to increase sample sizes and shared learning.
• Provide accessible data to support advocacy and policy discussions.

Getting involved in policy and advocacy

You do not need specialist policy knowledge to contribute. Small, regular actions can help influence systems and improve access to care over time.

• Stay informed through trusted patient organisations.
• Respond to public consultations on rare disease strategies.
• Use agreed, evidence-based messages when speaking with decision makers.
• Join awareness events and campaigns.

Helping through time, skills or donations

• Volunteer with patient organisations.
• Fundraise for research or family support services.
• Offer professional skills such as IT, design, legal or communications support.
• Support other families through peer networks and moderated groups.