National Institutes of Health and Other Agencies Research Initiatives Relevant to Barth Syndrome

In addition to vast investigator-initiated research that is supported by the National Institutes of Health (NIH) in the US, research in some specific areas is solicited by various NIH institutes from time to time. Applications for these are usually accepted three times a year. The following ongoing NIH initiatives are particularly relevant to Barth syndrome.

The Harrington Project for Discovery & Development

  • Deadline for Full Application: May 10, 2018

The Harrington Scholar-Innovator Award recognizes physician-scientist innovators throughout the U.S. whose research has the potential to advance standard of care. The Scholar-Innovator Award provides research and drug development funding as well as non-financial support to selected scholars and their institutions to help bridge the gap between basic discovery and clinical introduction.

The Harrington Scholar-Innovator Award selection committee seeks breakthrough discovery defined by innovation, creativity, and potential clinical impact including:

  • Discoveries deemed to address unmet medical needs
  • Novel targets
  • Potential to be developed into a commercial program
  • Possess intellectual property (IP) or protection strategy
  • May be either a small molecule, biologic, or other therapeutic modality
  • Diagnostics or devices only acceptable as part of a therapeutic development project or if completely transformative

Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01)

Funding Opportunity Announcement (FOA) Number: RFA-FD-16-043

  • Open Date (Earliest Submission Date): August 15, 2016 for October 14, 2016 application due date.
  • Open Date (Earliest Submission Date): August 15, 2018 for October 15, 2018 application due date
  • Letter of Intent Due Date(s): August 31, 2016 for October 14, 2016 application due date.
  • Letter of Intent Due Date(s): August 31, 2018 for October 15, 2018 application due date.
  • Application Due Date(s): October 14, 2016 and October 15, 2018, by 11:59 PM EST

Purpose: The objective of FDA's Orphan Products Natural History Grants Program is to support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics. The ultimate goal of these natural history studies is to support clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for natural history studies that will either assist or substantially contribute to market approval of these products.

Children's Cardiomyopathy Foundation

The Children´s Cardiomyopathy Foundation (CCF) offers two annual grant programs to support innovative basic, clinical, population, or translational studies relevant to the cause, diagnosis, or treatment of cardiomyopathy (dilated, hypertrophic, restrictive, left ventricular non-compaction, or arrhythmogenic right ventricular cardiomyopathy) in children under the age of 18 years. The goal of CCF´s grant programs is to advance medical knowledge of the basic mechanism of the disease and to develop more accurate diagnostic methods and improved therapies for children affected by cardiomyopathy.

Request for Research Proposals on Pediatric Cardiomyopathy

  • Letter of Intent Due Date: June 13, 2018 by 5:00 pm ET
  • Application Due Date: September 7, 2018 by 5:00 pm ET

American Society of Hematology

Patient Group Research Grant Opportunities

To draw together the multitude of hematology-related research grant opportunities that are available through patient groups, the Society has created a section on the ASH Web site that simplifies your search for requests for blood and blood disease research topics.

United Mitochondrial Disease Foundation

The United Mitochondrial Disease Foundation (UMDF) Research Grant Program began in 1997 out of a desire to fund research toward diagnoses, treatments, and cures for mitochondrial disease.

UMDF Research Grant Program will launch on Monday, March 20, 2017. After a period of internal evaluation, we have decided to make several changes to the UMDF Research Grant Program. Here are a few of the key changes:

  • Targeted Request for Proposal vs. General Solicitation: The Foundation will utilize a “Priority Topic” of BIOMARKER DEVELOPMENT AND VALIDATION in this cycle
  • Focus on Mentored Career Training and Young Investigator Awards: Program will fund Postdoctoral Fellowships as well as New and Early Stage Principal Investigators
  • Fixed Budget Amounts: Postdoctoral Fellowships and PI awards will have fixed budgets of $100,000 USD
  • Mitochondrial Disease-Focused Small Clinical Studies: Principal Investigators at any stage of career development will be eligible for this $25,000 USD funding mechanism

Streamlined Submission Process: Applicants will be invited to directly submit a Full Proposal - there will no longer be a Letter of Intent round

Compressed Timeline: Applicants will have 5 weeks to respond to the RFP when it is published on March 20, 2017 and awards will be announced in June 2017.

Peer Review, the hallmark of UMDF’s Research Grant Program for the past twenty years, will remain a critical component of the process.  
As per in previous cycles, all proposals will be created and submitted via our online portal:

Those researchers who have not yet registered on the portal are encouraged to do so immediately. New registrants (as well as researchers who already have an account) when logging in will be directed to create a new proposal as either a Postdoctoral Fellow or a Principal Investigator. Note, however, that you will not be able to progress the proposal you create until the grant cycle opens. As soon as the RFP for the March 2017 cycle is finalized it will be accessible from the main proposal page.

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