Center for Orphan Disease Research & Therapy

Rare Disease Overview CODRT Opportunities

RFA-Improved Therapies for Rare Diseases - MDBR Pilot Grants and Postdoctoral Fellowships

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 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.

Pilot grants are available for research related to: Adrenoleukodystrophy (ALD), Congenital Hyperinsulinism (CHI), Lymphangioleiomyomatosis (LAM), Mucolipidosis Type IV, Niemann Pick Type C (NPC), Pitt Hopkins, and RASopathies

Postdoctoral Fellowships are available for research related to: Congenital Muscular Dystrophy (CMD), Homozygous Familial Hypercholesterolemia (HoFH), Friedreich's Ataxia (FRDA), Hemophilia, Mucopolysaccharidosis (MPS), Niemann Pick Type C (NPC), and Tay-Sachs and related disorders

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

     RFA Posted: August 1, 2014

     Pre-application deadline: September 15, 2014, documents are to be uploaded no later than 5pm - PRE-APPLICATION IS CLOSED!

     Invitation to submit a Full-application by September 23, 2014

     Full-application deadline: October 20, 2014 (by invitation only) documents are to be uploaded no later than 8PM EST

     Anticipated start date: December 1, 2014


                         Postdoctoral Fellowship

                                     Postdoctoral Fellowship Application Guidelines

                                     Postdoctoral Fellowship Cover Page/Institutional Signature Form

                                     Postdoc online application

                        Pilot Grant

                                     Pilot Grant Application Guidelines

                                     Pilot Cover Page/Institutional Signature Form

                                     Pilot online application

                       Budget template

                       NIH Biosketch sample

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