At the Muscular Dystrophy Association’s biennial scientific conference, held in Washington, DC, on March 19-22, 2017, researchers from Acceleron Pharma presented a poster about the most prominent symptoms and daily life impact of FSH muscular dystrophy, as reported by patients and caregivers. The report was based on results from a survey developed by Acceleron in collaboration with Dr. Jeffrey Statland of the University of Kansas Medical Center and June Kinoshita from the FSH Society. Researchers at aTyr Pharma also contributed comments on the survey design.
The survey was sent to FSH Society members in December 2016, and 388 individuals responded. The most highly reported regions of muscle weakness were the scapula and abdominals, followed by biceps, ankle, and face. Leg, arm, and core weakness were considered to have the greatest impact on patients’ quality of life.
The survey respondents showed an extraordinarily high level of engagement, with 31 percent saying they had previously participated in a clinical research study. They are an altruistic group too. Some 69 percent said they would volunteer for a clinical trial even if it might not help them, because it might help others in the future.
“It’s really motivating to work alongside such a committed and selfless community of patients,” said Kinoshita. “They inspire all of us at the FSH Society to work extra hard.”
Click to download: MDA 2017 Poster – FSHD Patient Survey.