Author(s): Karen Dolan
We realize that we have been reactive rather than proactive, responding to requests rather than requesting proposals for research projects that we have identified as important. We hope to remedy this with a Think Tank – a meeting with key clinicians and researchers who will review current treatment strategies, identify potential novel approaches, and consider research needed to close the gap between what we now know and what we need to know to advance care.
We have been very fortunate in that the University of Pennsylvania’s Orphan Disease Center has offered to partner with us in this endeavor. They will be hosting a meeting of key researchers and clinicians on June 20, 2019, which MSUD Family Support Group President Sandy Bulcher and I will attend.
We look forward to this opportunity to learn from the attendees and advance research in a meaningful way.