Center for Orphan Disease Research & Therapy

Rare Disease Overview CODRT Opportunities

Grants and Pilot Projects

As a result of the Million Dollar Bike Ride, the Center for Orphan Disease Research and Therapy announces a Request for Applications (RFA) to support research from fundraising efforts and matching funds for $50,000 - $65,000 Pilot Grants, and $35,000 Postdoctoral Fellowship Grants.  International applications are welcome; the RFA is not restricted to Penn/CHOP or USA researchers.

The type of grant, pre and full application instructions, and scientific requirements are available by clicking on the following diseases: Adrenoleukodystrophy (ALD) - Pilot Grant , Congenital Hyperinsulinism (CHI) - Pilot Grant, Congenital Muscular Dystrophy (CMD) - Postdoctoral Fellowship, Homozygous Familial Hypercholesterolemia (HoFH) - Postdoctoral Fellowship, Friedreich's Ataxia (FRDA) - Postdoctoral Fellowship, Hemophilia - Postdoctoral Fellowship, Lymphangioleiomyomatosis (LAM)-2-Pilot Grants, Mucolipidosis Type IV - Pilot Grant, Mucopolysaccharidosis (MPS) - Postdoctoral Fellowship,  Niemann Pick Type C (NPC) - Postdoctoral Fellowship, Niemann Pick Type C (NPC) - Pilot Grant, Pitt Hopkins 2- Pilot Gants, RASopathies - Pilot Grant, and Tay-Sachs and related disorders - Postdoctoral Fellowship.

The timeline for the RFA/grant program is as follows:

Time Lines:

RFA Release Posted: August 1, 2014

Pre-application deadline: September 15, 2014, documents are to be uploaded no later than 5pm


Invitation to submit a Full-application by September 18, 2014

Full-application deadline: October 15, 2014 (by invitation only) documents are to be uploaded no later than 5pm

Anticipated start date: December 1, 2014

All applicants must become a member in the Center for Orphan Disease Research and Therapy to participate in this RFA. Please view our membership details.

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.

Mucopolysaccharidoses (PDF)

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