A new global alliance for Sanfilippo syndrome

05 Feb 2024

We are pleased to announce the formation of the International Sanfilippo Syndrome Alliance (ISSA), a global collaboration of patient organisations committed to the rapid delivery of benefits and solutions for individuals with Sanfilippo syndrome and their families.

The founding members of ISSA are eleven foundations from ten countries who have worked collaboratively on a range of initiatives, including co-funding research projects around the world. Formalising this alliance will help create a stronger, united voice focused on Sanfilippo syndrome. We acknowledge the many Sanfilippo families and organisations that have blazed a trail before us and the strong partnerships they have formed. So many families and their supporters have fought, over decades, to make a difference for their children and for children and families in the future. Without their work, Sanfilippo syndrome would not have received the attention it has and the Sanfilippo research community would not be the strong, diverse field that it is today.

Now, ISSA members seek to bring together the global Sanfilippo community to build on this incredible legacy and strive to achieve even faster, more efficient progress.While ISSA will be a global collaboration of patient organisations from around the world, each member will continue to operate individually on their respective missions. However, by collaborating on shared priorities for the whole community we can work more effectively with our colleagues in academic, clinical, not-for-profit, industry and regulatory communities to accelerate drug development and care solutions for Sanfilippo patients and their families.

We can support each other in advocacy to drive improvements in diagnosis, clinical care and family support, so that individuals with Sanfilippo can access the best care and support no matter where they live. We hope to share resources and expertise between the foundations and focus attention on the areas that will benefit from a globally coordinated approach.

Among our early priorities are the exploration of a patient-centred Sanfilippo registry and the translation and dissemination of shared resources, including the Global Roadmap for Sanfilippo Syndrome Therapies and the Sanfilippo Syndrome Consensus Guidelines for Clinical Care. We look forward to welcoming more members to ISSA as our work progresses, to raise all of our voices even higher.

For more information or to get involved please visit www.sanfilippoalliance.org or email the ISSA Secretariat at info@sanfilippoalliance.org.