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Simon Jones speaking at an MPS Society conference.

Expert event published on

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

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Hosted by the MPS Society (UK) in association with the International MPS Network (IMPSN), this hybrid event for professionals will be chaired by Prof Simon Jones and Prof Maurizio Scarpa.

Developing treatments for individuals with progressive neurocognitive or neuronopathic diseases such as Mucopolysaccharidosis (MPS) presents several significant challenges. Biomarkers can, however, play a critical role in assessing the efficacy of treatments, by providing objective measures of disease progression or response to therapy.

The aim of this meeting is to bring clinical and scientific experts, together with regulators, decision makers, commissioners, industry and patient organisations to facilitate the global sharing of information and discussion and to highlight the importance of integrating biomarkers into regulatory frameworks.

This is a hybrid meeting, attend in person or online.

Invitation for professionals, including:

  • Regulators

  • Country specific decision makers

  • Medics

  • Industry

  • Patient group representatives

  • HTA bodies

  • Commissioners

Proposed agenda

09:00 Welcome

09:10 Introduction

PART 1: CLINICAL AND PATIENT PERSPECTIVE

09:20 Clinical description of MPS

09:30 What about the neurology?

09:40 The uncertain path towards approval (patient perspective / post-trial perspective)

10:10 Clinical trials need biomarkers

10:20 Discussion

10:30 Break

PART 2: WHAT DOES THE SCIENCE TELL US?
(Rationale for Heparan sulfate as a biomarker)

10:50 Development of heparan sulfate assays

11:00 Gathering evidence to support heparan sulfate as a potential surrogate marker

11:10 The role of heparan sulfate in advancing clinical trial development

11:20 Impact of trial failure on clinicians and patients

11:30 Discussion

11:45 Lunch

Part 3: CURRENTLY ACCEPTED AND EVOLVING EVIDENCE

12:45 Clinical trial updates      

13:45 Discussion

PART 4: VIEWS FROM REGULATORS  

14:05 Global regulatory view

14:20 Reagan-Udall Foundation presentation

14:35 FDA presentation

14:50 Break

15:10 EMA presentation

15:25 MHRA presentation

15:40 Discussion

PART 5: PANEL DISCUSSION & RECOMMENDATIONS

16:00 Panel discussion & recommendations

16:20 Closing remarks

16:30 End

NB.:Topics and timings in the above draft agenda are subject to change.

Booking form

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