Collaboration opportunities in Hurler syndrome (MPS I-H) research
Progress in severe MPS I (Hurler syndrome) relies on strong collaboration between academic researchers, clinical teams, patient organisations and industry partners. This page highlights practical routes for collaboration across discovery science, translational research, clinical studies, registries and education.
Use this resource to identify partnership opportunities, align your work with existing initiatives and connect with others advancing MPS I-H research globally.
The case for collaborative research
Hurler syndrome is ultra-rare, biologically complex and demanding to study. Individual centres cannot address all research questions independently. Collaborative research enables:
- Access to sufficient participant numbers for robust preclinical and clinical analysis
- Pooling of specialist expertise across biology, gene therapy, HSCT, ERT, imaging and neurocognition
- Development of shared protocols, outcome measures and registry standards
- More rapid translation of laboratory findings into early-phase studies
Basic and translational science partnerships
Preclinical research in MPS I-H benefits significantly from shared resources, aligned methods and cross-laboratory validation.
- Coordinated use and harmonisation of validated animal models and endpoints
- Joint development of gene delivery platforms and CNS-targeting strategies
- Shared in vitro and ex vivo systems for vector testing and safety assessment
- Standardised assays for GAG measurement, imaging and composite outcomes
Preclinical tools & models · Vector design & mechanism · Efficacy outcomes
Shared cohorts, follow-up and outcomes research
Clinical collaboration addresses the challenges posed by small patient populations and long-term disease trajectories.
- Cross-centre analyses of outcomes following HSCT and ERT
- Joint studies of cardiac, skeletal, respiratory and neurocognitive progression
- Harmonised registry datasets with agreed core variables and timelines
- Multi-country health services and quality-of-life research
Follow-up & registries · Long-term outcomes · Living with Hurler syndrome
Statistics, modelling and health economics
- Bayesian and adaptive trial designs for small populations
- Integration of biomarkers and multi-domain outcomes
- Real-world evidence and cost-effectiveness modelling
- PK/PD translation and trial simulation approaches
Training and capacity building
- Joint PhD, MD/PhD and fellowship programmes
- Shared teaching materials and case-based learning
- Cross-institution mentorship and exchange schemes
- Webinars and collaborative teaching initiatives
How to propose collaboration
Clear, focused proposals help collaboration discussions progress efficiently. When contacting the team, please include:
- Your institution, role and specialist expertise
- The MPS I-H research area you are working in
- A brief outline of your project and current stage
- The type of collaboration you are seeking
Use the main contact route on hurlersyndrome.org (once available) and clearly indicate that your enquiry relates to research collaboration.
Contact usCollaboration opportunities at a glance
For researchers overview
Audience, themes and core research resources.
Research priorities
Key questions across CNS, skeletal, cardiac and gene therapy.
Preclinical tools & models
Animal models, assays and laboratory systems.
Biomarkers & outcomes
Connecting molecular correction to clinical benefit.
Follow-up & registries
Long-term data and real-world evidence.
Clinical trials roadmap
From preclinical data to first-in-human studies.