The fourth prong in Cure SMA’s research strategy is clinical care research. We fund clinical care research to understand the issues that affect daily life for people with SMA, from breathing to nutrition, and to improve their quality of life today.
Clinical Care Standards
In 2007, the Journal of Child Neurology published the “Consensus Statement for Standard of Care in Spinal Muscular Atrophy” and the “Family Guide to Consensus Statement for Standard of Care in Spinal Muscular Atrophy.” These documents established general guidelines for the management of SMA.
Cure SMA’s clinical care research builds on these consensus statements, with evidence-based projects that demonstrate positive results for those currently living with SMA.
Our Clinical Care Improvements
We won’t stop working toward a world without SMA, but until we have a treatment and cure, we’ll do everything we can to improve quality of life for children and families affected by the disease today. The outcomes of Cure SMA-funded clinical care projects may include:
- Pilot studies which then serve as the basis for larger, multi-center studies in SMA.
- New family-focused care publications, including Cure SMA care series booklets.
- Peer-reviewed journal publications to influence insurance coverage.
Our Clinical Care Funding
Periodically, Cure SMA issues a request for clinical care proposals. All applications are reviewed by our Medical Advisory Council, using an NIH-like scoring system based on both scientific quality and relevance to the Cure SMA mission of improving care for all those affected by SMA.
In 2016, an RFP will not be issued while we focus on other clinical care projects, including the development of a network of clinical care centers that will collaboratively collect patient care data to answer questions and develop strategies for optimal clinical management of SMA.