Future Directions
Proven treatments for SMA may soon become a reality as we gain a better understanding of disease pathogenesis.
Clinical trials to assess compounds that increase SMN protein levels in cell and animal models have begun. In the meantime, a proactive management strategy can help to limit disease progression and optimize outcome.
Undoubtedly, intervening as early as possible in the disease process will prove most effective as additional therapeutic strategies are identified. Participation in clinical trials can provide patients and families with additional clinical care and monitoring that can help to ensure that proactive care is the rule, rather than the exception.
Participation is also vital to the community to help demonstrate the effectiveness of potential treatments. However, a careful consideration of the potential risks as well as the time commitment involved should be carefully considered in each case.
View a listing of clinical trials on SMA involving the University of Utah or the Project Cure SMA Investigators Network.
