#ConnectedTogether
#MPSFamily2021
The 35th Annual Family Conference, Connected Together, united 550 participants from 20 countries around the world. We recognize that we are stronger when we collaborate and learn from one another.
Four days of conference material across two weekends allowed us to host over 60 different sessions and presentations for families and professionals. In her keynote address, Mercedes Ramirez Johnson shared of the importance of shifting perspective and seeing joy and hope in the midst of trauma and pain. This message resonated throughout the course of the conference, bringing to light new options for treatment, care, clinical trials, and plans on the horizon to transform the lives of those with MPS and ML.
The foundational content and critical updates following the keynote address formed the base knowledge for information over the course of the conference. Summaries of the presentations are provided below. For registrants, sessions are now available on-demand to watch for the next month by entering the auditorium and clicking on the graphic to direct you to session recordings.
Camp Courage, our programming for children, was a great undersea adventure! There were many fun activities the kids enjoyed over the course of the four days. Highlights included dance parties, scavenger hunts, playing games, acting out sea creatures and animals, drawing a mermaid, learning magic tricks from Captain Jack Spareribs, meeting an alligator and an iguana, and listening to stories from Auntie Angela.
In the MPS and ML Overview, Dr. Joseph Muenzer reviewed these diagnoses, and provided background information about the diseases. He reviewed current therapy options (including enzyme replacement therapy for MPS I, II, IVA, VI, and VII and hematopoietic stem cell transplantation for MPS I), and the limitations around these. He discussed approaches to managing challenging clinical symptoms and introduced some of the clinical trials and areas for consideration in treatment.
Dr. Barbara Burton, Dr. Matthew Ellinwood, and Dylan Simon with the Everylife Foundation discussed the process of newborn screening with the community. Sessions explored the process for adding a condition to the Recommended Uniform Screening Panel (RUSP) and for adoption by each state. The National MPS Society’s nomination of MPS II, Hunter syndrome, to the RUSP was discussed, and this is currently under evidence review.
Dr. Paul Harmatz provided information about some of the historical approaches to care and the onset of clinical trials for MPS. He discussed current trials and considerations for families, as well as current clinical care of patients. Dr. Lynda Polgreen discussed skeletal and joint pathology in MPS and how hematopoietic cell transplant and/or enzyme replacement therapy impacts bones and joints.
Dr. Elsa Shapiro and Dr. Julie Eisengart discussed neurocognitive and behavioral impacts of MPS, and considerations over time. They reviewed common symptoms present in conjunction with these conditions, and strategies for management and support. Dr. Shapiro explored the divergence of the expected developmental trajectory for those with MPS, and recommendations for continued study. Dr. Eisengart presented information about neurocognitive complications and clinical management and support strategies to assist individuals with MPS and their families.
Dr. Klane White discussed the orthopedic management of the mucopolysaccharidoses and reviewed common areas of concern in orthopedics across syndrome types. He explained areas of consideration for surgical intervention and decision-making to identify the appropriate processes and indicators for such. Dr. Paul Orchard provided information about bone marrow transplantation and updates for MPS disorders, the benefits of hematopoietic stem cell transplantation for MPS I, and future implications.
On Sunday, June 27, the conference started with family stories from the Bohley (MPS I) and Newman (MPS III) families, sharing their experiences of hosting fundraisers and making an impact in their communities. Dr. Barbara Burton explained genetics of MPS and ML and options for family planning. Each syndrome type then followed specific tracks for the remainder of the conference, joining together in Zoom breakout sessions to discuss information presented and symptoms unique to each diagnosis. Dr. Kim Hemsley provided a live presentation to MPS III families, and Dr. Paul Harmatz participated in the session for individuals with MPS VI and their families.
