The MSUD Family Support Group is teaming with the Clinic for Special Children in Strasburg, PA to conduct a Quality of Life Survey for MSUD families.
There is an urgent need and readiness across patients, drug developers and regulators to understand the critical needs of MSUD patients and caregivers. With this knowledge, clinical endpoints (what researchers should measure when conducting studies) can be identified. The Quality of Life Survey is a crucial missing piece, needed to synthesize these perspectives, resources, and energies to inform therapeutic development and review in this rare and devastating disease.
The objectives of the Quality of Life Survey are to:
- Understand the many ways in which MSUD affects the quality of life of MSUD families with the aim of developing drug and other treatments which will have the most impact.
- Identify potential research “end points” or metrics which can be incorporated into the development of MSUD registries, natural history studies, and future research project goals.
The survey will ask patients, parents (or other caregivers), and siblings to rate their experiences of how MSUD affects their day to day life. Questions will focus on:
- The physical effects of MSUD on patients and families, including questions about the MSUD formula,
- The mental stresses caused by the day to day management of MSUD,
- Social interaction issues, and
- The impact of the severity of MSUD on the patient and family member.