Center for Orphan Disease Research and Therapy
Areas of Rare Disease Focus for the Center
Based on the existing strengths within the Penn and CHOP communities, we have identified five initial areas of rare disease focus for the Center and five individuals to advise and help coordinate research and development in these areas. We anticipate that more focus areas will be added as the Center evolves.
- Neuromuscular Disease - H. Lee Sweeney, Ph.D.
- Metabolic Disorders - James M. Wilson, M.D., Ph.D.
- Ocular Disorders - Jean Bennett, M.D., Ph.D.
- Hematological Disorders - Katherine A. High, M.D.
- Rare Cardiovascular Diseases - Daniel J. Rader, M.D.
Center-Sponsored Research Projects on Rare Diseases for Members
To date, the Center has awarded over $2.8 million dollars to researchers on rare diseases. So far these grants have focused on lysosomal storage diseases, but in the future we plan to broaden the research awards to other rare disease areas as resources permit. These awards have not been limited to the Penn/CHOP community, as the Center strives to support rare disease efforts nationally and internationally. We plan to continue funding rare disease projects and expand to other rare diseases.
To gain access to funding opportunities, please click on Grants and Pilot Projects. These funding opportunities and other resources are only are available to members of the Center. Please click on the Membership page to join our Center.
Animal Testing Cores
Preclinical testing cores, that will maintain animal models of rare diseases, are being developed to support the disease focus areas of the Center. As Center supported Cores are developed, information on the services they provide and how to get access will be posted on this site.
RFA-Improved Therapies for MPS I (Award Is Closed to New Applications)
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.
All applicants must become a member in the Center for Orphan Disease Research and Therapy to participate in this RFA. Please click here for membership details.
Budget and Duration: Grants will be awarded for 1 to 2 years at $150,000 direct costs per year (10% indirect cost rate).
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.
Links to Outside Funding Opportunities
- Cicatricial Alopecia Research Foundation Closes:03/15/2014 Maximum:$20,000
- Cystinosis Foundation Deanna Lynn Potts Scholarship Closes:03/30/2014 Max:$1,000
- Myocarditis Foundation Research Grants Closes:12/15/2013 Maximum:$35,000
- Progeria Research Foundation Research Grants Closes:11/01/2013 Maximum: Inquire with funder
- Shwachman-Diamond Syndrome Foundation Closes:03/12/2014 Maximum:$35,000
- Willis-Ekbom Disease Foundation announcing the availability of three, $25,000 grants and a proposal deadline of December 1st.
- 2014 American Association for Dental Research Sjogren’s Syndrome Foundation Student Fellowship Closes:10/08/2013 Maximum:$4,000
- 2014 American Partnership for Eosinophilic Disorders HOPE Pilot Grant Closes:10/01/2013 Maximum:$50,000
- American Academy of Neurology/American Brain Foundation/Potamkin Family Closes:10/30/2013 Maximum:$100,000
- Sjogren’s Syndrome Foundation Closes:02/01/2014Maximum:$100,000
Links to Outside Resources
- National Organization for Rare Disorders
- NIH– ORDR – Office of Rare Disease Research
- FDA- Office of Orphan Products Development Funding Source
- Rare Disease Legislative Advocates