The Christopher Project is a partnership between patient advocacy organizations, health care providers/academic medical centers, and patients living with myotonic dystrophy, designed to determine North American patient and family member experiences with DM. Results from the cross-sectional, survey-based study have recently been published in Muscle and Nerve (Hagerman et al., 2019). The affected individual survey included 156 questions assessing DM demographics, diagnosis, symptoms, daily activity challenges, healthcare, insurance, treatments and interventions, and access to information and resources. The family member/caretaker survey included 97 questions regarding the symptoms and daily life activities of their family member or person under their care. DM type was self-identified. Results were reported from 1,180 patients and 402 family members/caregivers who responded to the survey.
Taken together, data from the Christopher Project helps to define unmet needs of patients living with DM and their family members or caretakers. These data are vital for development and design of clinical trials, clinical management strategies, and public health care policy.
The Myotonic Dystrophy Experience: A North American Cross-Sectional Study.
Hagerman KA, Howe SJ, Heatwole CE; Christopher Project Reference Group.
Muscle Nerve. 2019 Jan 24. doi: 10.1002/mus.26420. [Epub ahead of print]