This symposium is not accredited.
*Breakfast will be provided for those attending the sponsored breakfast session only.

Speaker: Dr. Philippe Campeau

This presentation will provide an overview of current and emerging therapeutic approaches for mucopolysaccharidoses (MPS), with a focus on long-term treatment outcomes and future innovations in care. Participants will review evidence related to the long-term efficacy of therapies for MPS IVA and MPS VI, including clinical outcomes and ongoing management considerations. The session will also explore emerging therapies, including novel substrate reduction therapies, brain-penetrating enzyme approaches, and gene therapy strategies aimed at improving disease management and patient outcomes. In addition, attendees will learn about advancements in gene therapy research for MPS-IIID, including recent findings and developments from Dr. Campeau’s laboratory.

At the end of the session, participants will be able to:

• Evaluate treatment efficacy in MPS IVA and MPS VI.
• Differentiate the clinical features of MPS IVA and MPS VI.
• Compare and prioritize different treatment modalities for MPS IVA and MPS VI.
• Describe emerging and future therapies for MPS IIID, MPS IVA, and MPS VI.
• Develop diagnostic and management approaches for patients with MPS IIID, MPS IVA, and MPS VI.