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.


Novel Approaches for Relating Genetic Variation to Function and Disease (R01 Clinical Trial Not Allowed)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PA-18-868
Open Date (Earliest Submission Date): September 5, 2018
Letter of Intent Due Date(s): Not Applicable
Application Due Date(s): Standard dates apply, by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates.
AIDS Application Due Date(s): Not Applicable
Expiration Date: July 6, 2021

Purpose:

Genome-wide association studies and other disease studies have found many variants that are statistically associated with disease risk, disease protection, or other traits. However, such studies do not show which variants in genomic elements cause these effects, or how they result in differences in function. Similarly, clinical genomic sequencing studies have identified many variants in healthy and diseased individuals. However, the pathogenicity of such variants is often unknown, leading to their classification as variants of uncertain significance (VUS), which makes clinical implementation difficult. This program aims to support the development of novel and generalizable approaches to study how genetic variants lead to differences in function and to study how such functional differences affect human health and disease processes or how this knowledge can be used clinically.


Novel Approaches for Relating Genetic Variation to Function and Disease (R21 Clinical Trial Not Allowed)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PA-18-867
Open Date (Earliest Submission Date): September 16, 2018
Letter of Intent Due Date(s): Not Applicable
Application Due Date(s): Standard dates apply, by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates.
AIDS Application Due Date(s): Not Applicable
Expiration Date: July 17, 2021

Purpose:

Genome-wide association studies and other disease studies have identified many variants that are statistically associated with disease risk, disease protection, or other traits. However, such studies do not generally show which specific variants in genomic elements cause these effects, or how they result in differences in function. Similarly, genomic sequencing studies in clinical settings have identified many variants in healthy and diseased individuals. However, the pathogenicity of such variants is often unknown, leading to their classification as variants of uncertain significance (VUS), which makes clinical implementation difficult. This Program Announcement and the companion R21 Program Announcement aim to support the development of novel and generalizable approaches to study how genetic variants lead to differences in function and to study how such functional differences affect human health and disease processes or how this knowledge can be used clinically.


Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01)
Participating Organization(s): U.S. Food and Drug Administration
Funding Opportunity Announcement (FOA) Number: RFA-FD-19-001
Open Date (Earliest Submission Date): November 10, 2018
Letter of Intent Due Date(s): December 10, 2018
Application Due Date(s): January 10, 2019 by 11:59 PM Eastern Time
Expiration Date: January 11, 2019

Purpose:

The purpose of this funding opportunity announcement (FOA) is to support studies that advance medical product development through the characterization of the natural history of rare diseases/conditions with unmet needs. Through the support of efficient and innovative natural history studies, FDA expects to address critical knowledge gaps, to remove major barrier(s) to progress in the field, to exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and to inform current or future product development including the design of clinical trial(s) and to ultimately inform the development of medical products that meet patient needs. 


Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R03 Clinical Trial Not Allowed)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PAR-18-952
Open Date (Earliest Submission Date): December 07, 2018
Letter of Intent Due Date(s): 30 days prior to the application due date
Application Due Date(s): January 7, 2019, June 10, 2019, October 24, 2019, June 8, 2020, October 26, 2020, June 7, 2021, October 25, 2021 , by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates.
Expiration Date: October 26, 2021

Purpose:

This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare disease research by enabling efficient and effective movement of candidate therapeutics or diagnostics towards clinical trials, and to increase their likelihood of success through development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.


Clinical Trial Readiness for Rare Diseases, Disorders, and Syndromes (R21 Clinical Trial Not Allowed)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PAR-18-953
Open Date (Earliest Submission Date): December 07, 2018
Letter of Intent Due Date(s): 30 days prior to receipt date
Application Due Date(s): January 7, 2019, June 10, 2019, October 24, 2019, June 8, 2020, October 26, 2020, June 7, 2021, October 25, 2021,  by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates.
AIDS Application Due Date(s): Not Applicable
Expiration Date: October 26, 2021

Purpose:

This Funding Opportunity Announcement (FOA) invites researchers to submit applications for support of clinical projects that address critical needs for clinical trial readiness in rare diseases. The initiative seeks applications that are intended to facilitate rare disease research by enabling efficient and effective movement of candidate therapeutics or diagnostics towards clinical trials, and to increase their likelihood of success through development and testing of rigorous biomarkers and clinical outcome assessment measures, or by defining the presentation and course of a rare disease to enable the design of upcoming clinical trials.


Research Project Grant (Parent R01 Clinical Trial Required)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PA-19-055
Open Date (Earliest Submission Date): January 5, 2019
Letter of Intent Due Date(s): Not Applicable
Application Due Date(s): Standard dates apply, by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates. The first standard application due date for this FOA is February 5, 2019.
AIDS Application Due Date(s): Standard AIDS dates apply by 5:00 PM local time of applicant organization. All types of AIDS and AIDS-related applications allowed for this funding opportunity announcement are due on these dates. The first AIDS application due date for this FOA is May 7, 2019.
Expiration Date: January 8, 2022

Purpose:

The NIH Research Project Grant supports a discrete, specified, circumscribed project in areas representing the specific interests and competencies of the investigator(s). This Parent Funding Opportunity Announcement requires that at least 1 clinical trial be proposed. The proposed project must be related to the programmatic interests of one or more of the participating NIH Institutes and Centers (ICs) based on their scientific missions.

Applicants should note that some ICs (see Related Notices) only accept applications proposing mechanistic studies that meet NIH's definition of a clinical trial through this funding opportunity announcement.


Research Project Grant (Parent R01 Clinical Trial Not Allowed)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PA-19-056
Open Date (Earliest Submission Date): January 5, 2019
Letter of Intent Due Date(s): Not Applicable
Application Due Date(s): Standard dates apply, by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates. The first standard application due date for this FOA is February 5, 2019.
AIDS Application Due Date(s): Standard AIDS dates apply by 5:00 PM local time of applicant organization. All types of AIDS and AIDS-related applications allowed for this funding opportunity announcement are due on these dates. The first AIDS application due date for this FOA is May 7, 2019.
Expiration Date: January 8, 2022

Purpose:

The NIH Research Project Grant supports a discrete, specified, circumscribed project in areas representing the specific interests and competencies of the investigator(s). The proposed project must be related to the programmatic interests of one or more of the participating NIH Institutes and Centers (ICs) based on their scientific missions.  

This Funding Opportunity Announcement does not accept applications proposing clinical trial(s)


Limited Competition for NIH-Industry Program: Discovering New Therapeutic Uses for Existing Molecules (U01 Clinical Trial Required)
Participating Organization(s): National Institutes of Health
Funding Opportunity Announcement (FOA) Number: PAR-18-910
Open Date (Earliest Submission Date): April 3, 2019
Letter of Intent Due Date(s): 30 days prior to receipt date.
Application Due Date(s): May 3, 2019, August 26 2019, May 4, 2020, August 26, 2020, May 3, 2021, August 26, 2021, by 5:00 PM local time of applicant organization. All types of non-AIDS applications allowed for this funding opportunity announcement are due on these dates.
AIDS Application Due Date(s): Not Applicable
Expiration Date: August 27, 2021

Purpose:

This Funding Opportunity Announcement (FOA) seeks applications that propose testing new therapeutic uses for experimental drugs or biologics (Assets) across a broad range of human diseases in adult and pediatric populations. This innovative program allows investigators to propose new therapeutic uses for Assets from pharmaceutical company partners. Strong applications will include scientific evidence that modulation of an Asset’s target will have a positive impact on the disease/condition. 


Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
Participating Organization(s): U.S. Food and Drug Administration
Funding Opportunity Announcement (FOA) Number: RFA-FD-20-001
Open Date (Earliest Submission Date): April 25, 2019
Letter of Intent Due Date(s): May 25, 2019
Application Due Date(s): June 25, 2019, by 11:59 PM Eastern Time
AIDS Application Due Date(s): Not Applicable
Expiration Date: June 26, 2019

Purpose:

The purpose of this funding opportunity announcement (FOA) is to support clinical studies of products that address unmet needs in rare diseases or conditions or provide highly significant improvements in treatment or diagnosis. Through the support of efficient and innovative clinical studies evaluating safety and/or effectiveness, FDA expects to increase the number of treatments for rare diseases with an unmet medical need and support studies that will either result in market approval of these products or substantially contribute to the essential data needed for medical product development that will ultimately meet the needs of rare disease patients.


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.

AHA/CCF Pediatric Cardiomyopathy Join Research Award

The Children's Cardiomyopathy Foundation (CCF) has partnered with the American Heart Association (AHA) to establish a research award for investigators conducting research specific to pediatric cardiomyopathy. Funding for the selected award will be provided equally by CCF and AHA. The deadline for the Transformational Project Award is approaching on January 23, 2019, and interested investigators are encouraged to submit their proposals on pediatric cardiomyopathy.

Purpose:

The Transformational Project Award, focused on pediatric cardiomyopathy, is a three-year award jointly funded by the AHA and CCF. The total award amount is $300,000. The award supports highly innovative, high-impact projects that build on work in progress that could potentially lead to critical discoveries or major research advancements. Proposals for the Transformational Project Award should represent the second phase of a research concept and include preliminary data. Visit AHA’s site for the program description and eligibility.

Interested parties should submit an application for one of the following research awards: Career Development, Innovative Project Award, or Transformational Project Award. For questions on applications please email: apply@heart.org.


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: www.umdf.fluidreview.com.

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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