Rare Disease Overview
The mucopolysaccharidoses (MPS) are a group of inherited lysosomal storage disorders. Lysosomes function as the primary digestive units within cells. Enzymes within lysosomes break down or digest particular nutrients, such as certain carbohydrates and fats. In individuals with MPS disorders, deficiency or malfunction of specific lysosomal enzymes leads to an abnormal accumulation of certain complex carbohydrates (mucopolysaccharides or glycosaminoglycans) in the arteries, skeleton, eyes, joints, ears, skin, and/or teeth. Read more
RFA-Improved Therapies for MPS I (Award Is Closed to New Applications)
MPS I RFA Improved Therapies August 1, 2013 (PDF)
Overview of the RFA Award
The Center for Orphan Disease Research and Therapy announces a Request for Applications (RFA) to support research on the development of improved therapies for patients with syndromes due to MPS I including Hurler, Hurler-Scheie and Scheie. Particular emphasis will be on treatments that improve aspects of the disease which are not adequately treated by enzyme replacement therapy such as pathology in the CNS, skeletal system, eye and heart among others. It is anticipated that most grants will utilize pre-clinical and clinical models of MPS I. However, applications will also be considered that utilize models of mucopolysaccharidoses other than MPS I if their use is scientifically justified and the data can be directly applied to improved therapies for MPS I.
- Request for Applications on Improved Therapies for MPS I (PDF)
- MPS I Grant Full Application Guidelines (PDF)
- MPS I Cover Page (PDF)
- All applicants must become a member in the Centerfor Orphan Disease Research and Therapy to participate in this RFA. Please view our membership details.
- Eligibility: All individuals holding a faculty-level appointment at an academic institution or a senior scientific position at a biopharmaceutical company are eligible to respond to this RFA.
- Budget and Duration: Grants will be awarded for 1 to 2 years at $150,000 direct costs per year (10% indirect cost rate).