好色先生

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Abstract Details

A Roadmap to Patient Engagement: FSHD and the ReSolve Clinical Trial
Research Methodology, 好色先生, and History
S23 - Research Methodology and 好色先生 (4:32 PM-4:40 PM)
004
To describe our efforts to overcome barriers to patient engagement in facioscapulohumeral muscular dystrophy (FSHD), as well as offer a roadmap that can be replicated in other rare neurological disorders.
Patient engagement can increase recruitment and retention and contributes to the relevance of the research. People with rare diseases are highly motivated to participate in research, but historically they have been involved exclusively as “subjects” rather than as research collaborators. Challenges for engagement in rare diseases include a lack of patient to patient interaction, geographic distance among patients and sites of care, and a lack of researcher training in how to conduct meaningful engagement. 
We implemented an engagement plan during Clinical Trial Readiness to Solve Barriers to Drug Development for FSHD (ReSolve), an 18-month, multi-site, observational study of individuals with FSHD. Elements of our engagement plan included conducting focus groups during protocol development, patient involvement on the ReSolve external advisory committee, creation of a patient advisory committee, and collaboration with patient advocacy groups. 
Patient feedback led to adaptations in the study protocol and to changes in recruitment and retention methods. Neurologists and study team members acknowledged the benefit of early and sustained patient engagement in the ReSolve study process. 
Patient engagement in research adds to its value and can increase recruitment and retention.  Our engagement efforts give an example of how collaboration with patients and families can be accomplished in FSHD and the co-developed roadmap process may be replicable in other rare neurological diseases. 
Authors/Disclosures
Samantha J. LoRusso, MD (Kaiser)
PRESENTER 		Dr. LoRusso has nothing to disclose.
Katy Eichinger, PhD, PT, DPT, NCS (University of Rochester) Dr. Eichinger has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Fulcrum. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Avidity. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for DyneTherapeutic. Dr. Eichinger has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Dr. Eichinger has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TRiNDS. The institution of Dr. Eichinger has received research support from Charcot Marie Tooth Association. The institution of Dr. Eichinger has received research support from Muscular Dystrophy Association. Dr. Eichinger has received intellectual property interests from a discovery or technology relating to health care.
No disclosure on file
Leann Lewis Leann Lewis has nothing to disclose.
Michaela Walker, MPH, CCRP (University of Kansas Medical Center) Miss Walker has nothing to disclose.
Alrabi Tawil, MD, FAAN (University of Rochester Medical Center) Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Tawil has received personal compensation in the range of $500-$4,999 for serving as a Consultant for meRicule. The institution of Dr. Tawil has received research support from Friends of FSH Research. The institution of Dr. Tawil has received research support from FSH Society. The institution of Dr. Tawil has received research support from NIH. The institution of Dr. Tawil has received research support from Fulcrum. Dr. Tawil has received intellectual property interests from a discovery or technology relating to health care.
Jeffrey Statland, MD (University of Kansas Medical Center) Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Arrowhead. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ML Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Epic Bio. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Armatus . Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kate Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Vita Therapeutics. Dr. Statland has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dyne Therapeutics. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity . Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fulcrum Therapeutics. Dr. Statland has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Statland has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex . The institution of Dr. Statland has received research support from NIH. The institution of Dr. Statland has received research support from FSHD Society. The institution of Dr. Statland has received research support from Friends of FSH Research. The institution of Dr. Statland has received research support from FSHD Canada. The institution of Dr. Statland has received research support from MDA.
No disclosure on file