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.

NCATS Rare Diseases Are Not Rare! Challenge

Notice Number: NOT-TR-19-002

Key Dates
Release Date: September 26, 2018
The Challenge begins: September 30, 2018 
Submission period: September 30, 2018 – October 28, 2018
Judging Period: November 12, 2018 – November 26, 2018
Winners Announced: December 2018


The purpose of this notice is to announce the NCATS Rare Diseases Are Not Rare! Challenge. The goal of this challenge is to reward and spur innovative approaches to effectively communicate with others and to educate people about rare diseases through social media or art. 

The total prize purse is up to $5,000 awarded as follows:

  • First place: $3000 and travel expenses for up to 4 people to participate in Rare Disease Day at NIH, February 28, 2019 to present the winning entry
  • Second place: $1500
  • Third place: $500
  • Honorable mentions (10) will be posted on NCATS’ website

For full details about eligibility requirements, competition rules, detailed judging criteria, and deadlines for submissions, please consult the NCATS Rare Diseases Are Not Rare! Challenge page.


The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health (NIH), is seeking innovative ways to communicate with others to educate people about rare diseases through social media or art. The goal of this challenge, which is being led by NCATS’ Office of Rare Diseases Research (ORDR), is threefold: First and foremost, it is to raise awareness for all rare diseases in a collective manner. Second, it is intended to bring attention to the many people with rare diseases; and finally, it is to highlight the need for research and the development of new treatments. You can help us get the word out by competing in our rare disease prize competition!

Find a way to communicate with others and to educate people about rare diseases through social media or art. Use any communication vehicle you choose; be as creative and original as possible. Here are examples of appropriate communication vehicles:

  • music video
  • song (with or without sheet music)
  • dramatic reading
  • poem
  • painting
  • mime
  • poster
  • comic
  • animation
  • photo/collage
  • new name for “rare diseases”
  • puppets

Each team or individual may submit only one entry.

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.

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.

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:

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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