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Role of biomarkers in advancing access to treatments for individuals with neurocognitive or neuronopathic MPS conditions

Hosted by the MPS Society UK in association with the International MPS Network (IMPSN), this meeting brought together a diverse group of voices and expertise from patient and carer representatives, clinical teams, scientists, industry professionals and regulators, all with one shared mission: to advance our understanding and treatment of MPS conditions and to highlight the importance of integrating biomarkers into regulatory frameworks.

The MPS Society and IMPSN would like to extend their gratitude to everyone who attended this pivotal meeting on Tuesday, March 18, 2025. For those unable to attend or who wish to revisit some of the excellent presentations, you can find the video recordings on this page in four parts. Further information, including a summary report and individual presentation slides will follow.

The meeting focused on the critical role of biomarkers in clinical trials, with a particular emphasis on heparan sulfate as a key example in the context of MPS. As many of you know, MPS is a complex group of diseases where neurological decline is often irreversible, making traditional clinical trial methods challenging when it comes to assessing treatment efficacy. This is where biomarkers like heparan sulfate become crucial, providing us with the opportunity to track disease progression, evaluate therapeutic impact earlier and ultimately intervene in a timely manner to improve patient outcomes.

Throughout the meeting, we delved into the scientific foundations, regulatory considerations and clinical applications of heparan sulfate, and explored how we can leverage this knowledge to improve the future for MPS treatments.

Download the programme

Part 1 Clinical & Patient perspective

Maurizio Scarpa and Simon Jones set the scene for the reasons behind the meeting followed by an overview of MPS conditions and the clinical and patient perspective for why clinical trials need biomarkers.