Research Priorities and Requests For Proposals
We are all united in our hope of improving the lives of those with MSUD. Promoting research directed at advancing treatments and discovering a cure is a primary goal of the MSUD Family Support Group.
Research can improve the lives of MSUD patients by:
- Discovering drugs which could lower blood leucine and keto-acid levels and/or reduce their toxic effects.
- Developing home monitoring devices to monitor blood amino acid levels.
- Developing gene therapies which could fix the defect in amino acid metabolism causing MSUD.
- Identifying the impact of MSUD on the brain and developing therapies to improve neuro-psychological health.
- Improving metabolic formulas.
- Identifying unmet needs of those with MSUD and their families.
Current research priorities:
The present funding priorities of the MSUD Family Support Group are to support research that will promote advances in the treatment and management of MSUD, with the long term goal of facilitating the development of a cure. The three top priorities at present are to:
- Support the development of an in-home technology which can measure blood leucine levels of MSUD patients.
- Understand the impact of MSUD on the brain and develop therapies to improve neuro-psychological health.
- Develop gene therapies which could fix the defect in amino acid metabolism causing MSUD.
We will, however, entertain any proposal which can be reasonably expected to advance treatments and improve the lives of those with MSUD.
The MSUD Family Support Group (MSUD-FSG) works to improve the lives of individuals with Maple Syrup Urine Disease (MSUD) and pursue a cure. The MSUD Research Fund is a campaign of the (MSUD-FSG) to advance the science of MSUD by funding the most promising research that will lead to new therapy discoveries and a cure. The MSUD-FSG seeks proposals that will help us meet the above strategic goals as well as close existing gaps in the knowledge and science of MSUD, and proposals to further support existing MSUD-related projects. Applicants are invited to submit project proposals meeting these goals using the guidelines presented in its Request For Proposals. For questions related to MSUD research, contact MSUD Family Support Group Research Lead Karen Dolins at firstname.lastname@example.org or 914-391-2982.