Funding opportunities in Hurler syndrome (MPS I-H) research
Progress in Hurler syndrome research relies on long-term, sustainable funding. This page is designed to support researchers, clinicians and partners in thinking strategically about how to secure funding for projects across the full research pathway, from basic science and preclinical development through to clinical trials, registries and quality-of-life studies.
Use this guidance to understand key funding routes, position MPS I-H within broader rare disease and gene therapy calls, and align applications with clearly defined priorities and unmet clinical needs.
The case for investment in Hurler syndrome research
Although Hurler syndrome is ultra-rare, its impact on individuals and families is substantial and lifelong. Dedicated investment enables the research needed to:
- Address ongoing unmet needs despite HSCT and enzyme replacement therapy.
- Develop and evaluate advanced therapeutic strategies, including systemic gene delivery.
- Improve long-term outcomes, quality of life and care pathways.
- Generate robust evidence to support equitable global access to diagnosis and treatment.
Framing proposals within this wider context is often critical to building a strong funding case.
Main categories of research funding
MPS I-H research is commonly supported through a combination of funding sources rather than a single route.
- Public and national research agencies supporting investigator-led science and clinical studies.
- International and rare disease consortia focused on gene therapy, paediatrics and registries.
- Charitable foundations and disease-specific organisations funding pilot and clinical work.
- Industry and public–private partnerships for translational and clinical programmes.
- Institutional seed funding, fellowships and early-career schemes.
Making your case compelling
Funders increasingly expect proposals to demonstrate clear links between research questions, unmet need and long-term impact. Alignment with established MPS I-H priorities strengthens applications.
- Targeting CNS involvement, skeletal disease, cardiopulmonary complications or survivorship.
- Building on existing HSCT, ERT or systemic gene therapy approaches.
- Integrating biomarkers, outcome measures and registry data.
- Embedding patient experience and quality-of-life perspectives.
Supporting basic science and gene therapy programmes
Preclinical and translational research is essential for reducing risk and accelerating innovation in MPS I-H.
- Use of robust, clinically relevant animal and cellular models.
- Innovative vector platforms, delivery strategies and biomarkers.
- Clear translational pathways towards first-in-human studies.
- Commitment to collaboration, sharing tools and data.
Supporting long-term follow-up and service improvement
Clinical and registry-based research provides essential insight into real-world outcomes following existing and emerging therapies.
- Well-defined hypotheses addressing neurocognitive, skeletal or cardiac outcomes.
- High-quality data collection and harmonised assessment approaches.
- Patient-centred and functional outcome measures.
- Clear potential to inform guidelines and service delivery.
Funding people as well as projects
Building expertise and research capacity in MPS I-H is a fundable objective in its own right.
- PhD and MD/PhD studentships across laboratory and clinical domains.
- Clinical fellowships with protected research time.
- Allied health and nursing research focused on holistic care.
- Structured supervision and cross-centre collaboration.
Charitable and philanthropic funding streams
Charities and philanthropic donors frequently support targeted, high-impact projects in rare diseases.
- Co-designed research with patient organisations.
- Pilot and seed studies to unlock larger funding.
- Educational resources and care pathway development.
- Transparent communication of risks and timelines.
Structuring academic–industry funding relationships
Industry partnerships can bring funding, expertise and infrastructure to translational research.
- Clear definition of scientific roles and responsibilities.
- Independence in data analysis and publication.
- Alignment with regulatory and evidence requirements.
- Strong patient-centred ethical frameworks.
Key components of a competitive application
- Focused aims linked to recognised research priorities.
- Strong rationale grounded in natural history and unmet need.
- Robust and realistic methodology.
- Multidisciplinary team with defined expertise.
- Patient and family engagement.
- Clear plans for dissemination and impact.
Turning ideas into funded projects
- Map projects to themes funders recognise, such as rare disease or gene therapy.
- Use institutional research offices for funding intelligence.
- Engage early with charities to understand priorities.
- Explore joint or co-funded applications.
Funding opportunities at a glance
- MPS I-H research is fundable across basic, clinical and QoL domains.
- Multiple funding routes exist, including public, charitable and industry sources.
- Strong alignment with priorities and outcomes strengthens applications.
- Training and capacity-building support long-term sustainability.
For researchers overview
Core themes and resources
Research priorities
Key questions for funding
Preclinical tools & models
Translational foundations
Biomarkers & outcomes
Meaningful endpoints
Follow-up & registries
Real-world evidence
Collaboration opportunities
Building partnerships