For families affected by neuronopathic mucopolysaccharidoses every second counts.
These are serious conditions that get worse over time, especially in the brain. Getting treatment early, before symptoms start or get worse, can make a huge difference. But too often, children are diagnosed too late to take part in clinical trials or access treatments that might help them.
Therefore, in March we hosted an expert meeting on the Role of biomarkers in advancing access to treatments for individuals with neurocognitive or neuronopathic MPS conditions together with the International MPS Network.
This multi-stakeholder meeting brought together clinical and scientific experts, regulators, decision makers, commissioners, industry and patient organisations to discuss the urgent need for ethical, effective and efficient clinical trial pathways in neuronopathic mucopolysaccharidoses (MPS).
Biomarkers can play a critical role in assessing the efficacy of treatments by providing objective measures of disease progression or response to therapy. The meeting focused on the role of heparan sulfate as a biomarker and disease-driving metabolite and the importance of integrating biomarkers into regulatory frameworks.
