PPMD will convene a pre-conference meeting of physical therapists, occupational therapists, and clinical coordinators who specialize in the research, assessment, longitudinal data collection, and treatment of people with Duchenne, June 25 and 26 in Chicago. This meeting will precede PPMD’s 20th Annual Connect Conference, June 26-29.
PPMD Founding President and CEO, Pat Furlong, explains the origin of the meeting:
For the last several years, PPMD has brought together experts in Duchenne care to discuss gaps we have found in the clinical system. Recently, we held meetings with several biotech and pharmaceutical companies to explore barriers to clinical trials. Our discussions focused on three major areas of concern: variability in care, the need for standardizing physical therapy evaluation of a child with Duchenne and recommendations for care, and the need for standardizing outcome measures for clinical trials. This year’s pre-conference meeting will bring together top therapists from clinics around the country and start what we hope will be an ongoing dialogue about standardizing outcome measures. We are blessed to be a rare disease with a pipeline full of potential therapies. Now we need to make sure there are uniform parameters in place so that clinical trials in Duchenne are as effective and efficient as possible.
The goals of this meeting are to establish a platform for dissemination and discussion of the most up-to-date information about Duchenne in an effort to coordinate care and standardize the collection of outcome measures. The agenda will include discussions of magnetic resonance imaging and spectroscopy, strength, function, and respiration. These discussions will lay a foundation for outcome measures in all areas.
PPMD is hosting this meeting with the help of Claudia Senesac, PT, PhD, PCS from the University of Florida (Gainesville). PPMD has a long-standing history with the University of Florida – first supporting the investigation of MRI as a potential outcome measure and expansion of natural history studies for Duchenne with “Imaging DMD.” PPMD initiated discussion with the University based on concerns expressed from meetings with industry partners. Dr. Senesac will help lead this effort to examine the nuances of measuring outcomes so that we can better standardize the way in which clinical trials are conducted.
This will be the first of several meetings that will provide training and standardization of outcome measures in Duchenne. PPMD will provide the Duchenne community, including therapists and clinicians unable to attend, with regular updates from these ongoing discussions.
To learn more about PPMD’s extensive care initiatives, visit PPMD’s website.