COMBINEDBrain News

FDA Grant Funding Opportunity for Rare Disease Research - Deadline is 10/20/26

COMBINEDBrain — Sat, 03 Oct 2026 22:43:27 GMT

Announcement of Grant Funding Opportunity for Rare Disease Research:

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce a new funding opportunity for the Orphan Products Grants Program to support clinical trials for rare diseases and conditions. The purpose of this funding opportunity announcement is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions.

Through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development.

Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)

Receipt Date: October 20, 2026

FOA Number: RFA-FD-25-020

• Purpose: To fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions.

• Focus: Efficiency, innovation, impact, collaboration, leveraging patient input, infrastructure and financial resources

• Efficiency: Encourage efficient and innovative clinical trials in all phases of product development through adaptive and seamless trial designs, basket, umbrella and platform trials studying multiple rare diseases/products and the use of data modeling and simulations. Allow additional total costs up to $250,000 per year for studies using efficient and innovative designs.

• Contact: Katherine Needleman, Director, Orphan Products Grants Program

E-mail: katherine.needleman@fda.hhs.gov

• Additional Information: OOPD Website

Please forward this announcement to any parties who may have need and interest in this grant program and please post the information on social media to alert interested parties.

Thank you for your help in bringing awareness to this important opportunity.

Bridget Nugent, Ph.D.

Health Scientist, Office of Orphan Products Development

Office of the Commissioner

U.S. Food and Drug Administration

U.S. Department of Health and Human Services

Tel: 301-796-9443

Inaugural Research Roundtable for Rare Genetic Neurodevelopmental Disorders is in the books!

COMBINEDBrain — Thu, 01 Oct 2026 21:34:05 GMT

From Terry Jo Bichell via LinkedIn:

What a historic 2 days!! The very first RR-RGND launched in Arlington, Virginia on Sept 28-29 and the right people were in the room when it happened!! All the stakeholders: Industry, Patient Advocates, Clinicians, FDA, Funders, Statisticians, Psychometricians, even Payers were in the room with one problem to solve: “Is the child's life better because they took the medication? And how do we measure that?” It is a notoriously thorny problem to measure benefit in people with cognitive disability who have a hard time speaking for themselves. The historic shift was for collaboration rather than competition between all these players to help get good treatments to kids who need them. I am so proud to have worked closely with Elizabeth Berry-Kravis, Michelle Campbell Emma James Stéphane Auvin, MD, PhD, FAES Konrad J. Werhahn Kimberly Goodspeed, MD Sunitha Malepati, Charlene Son Rigby, Annie Kennedy, Berit Powers, Laura Pisani, MD, MBA, FACMG, Nicola Williamson, Robert Komorowski, Scott Demarest, Bryce Reeve, Smitha Jagadish, Rachael Hawtin and the COMBINEDBrain team to make this meeting come together. It was built on the shoulders of giants, Jacqueline French and the Epilepsy Foundation Research Roundtable for Epilepsy.

THANK YOU, ANNUAL SUMMIT SPONSORS!

COMBINEDBrain — Tue, 08 Sep 2026 22:35:20 GMT

THANK YOU TO OUR GENEROUS ANNUAL SUMMIT SPONSORS!

There are more opportunities available! Click HERE to request information!

No Biorepository Roadshow and EEG Collection at CB Annual Summit!

COMBINEDBrain — Tue, 25 Aug 2026 20:00:06 GMT

We did not get the required 10 participants signed up for a roadshow at the Annual Summit so we won't be having one this year.

THANK YOU!

RARE: A Rare Disease Revolution - Now Streaming

COMBINEDBrain — Thu, 06 Aug 2026 19:28:44 GMT

From a soldier to a musician, from an immigrant family to a mother of twins, meet the unsuspecting heroes waging war against rare disease. Love drives change in this rare disease revolution, where parents become scientists and warriors who wage battles in labs, Congress, and their own homes to drive scientific breakthroughs, hope and cures for countless others. Rent the documentary here!

Children's Book Author Donates Profits to COMBINEDBrain

COMBINEDBrain — Thu, 23 Jul 2026 18:07:49 GMT

Paige Kalik, Genetic Counseling student, has authored a book about being the sibling of a child affected by a rare genetic neurodevelopmental condition. Emily Amerson, PAG Leader and Governing Board Member, illustrated it! Paige is donating profits from the sale of this book to COMBINEDBrain. You may order your copy here or at the QR code below!

Newly Launched Center for Therapeutic Genetics

COMBINEDBrain — Wed, 22 Jul 2026 15:55:18 GMT

Shared by Jared Bancroft, TPRM3 Foundation

Thought this would be of interest to folks here ...

Broad, Boston Children’s, and Jackson Labs have just launched the Center for Therapeutic Genetics. It is aimed at finding treatments for rare and ultra-rare diseases. One of their early areas of focus will be in CNS specifically around genetic epilepsies. Definitely something to keep an eye on as they get off the ground. Click the link above and go to the "Connect" tab to sign up for their newsletter to keep apprised of their progress!

Press release with more details -

https://www.broadinstitute.org/news/broad-institute-boston-childrens-hospital-and-jackson-laboratory-launch-center-therapeutic

Grant Opportunity Announcement - Deadline August 10

COMBINEDBrain — Mon, 20 Jul 2026 16:33:40 GMT

COMMUNITY HEALTH EQUITY GRANT OPPORTUNITY!

Apply to Our 2026 Grant Cycle by August 10th

RTW Foundation's 2026 grant cycle is still open! If your organization is committed to advancing health equity in New York City for communities of color, low-income neighborhoods, recent immigrants, and other underserved populations, be sure to submit your LOI before the deadline.

This year, our community health team is expanding services beyond funding to support communities and organizations particularly impacted by changes in safe, affordable access to care. We look forward to providing grant funding, co-hosting health fairs, and expanding health education with local health organizations.

LOI Deadline: Monday, August 10th - https://www.tfaforms.com/5226638

Learn more and submit an LOI here

Please feel free to share this opportunity with local health equity organizations in New York City. If you have any questions or want to connect, please reach out to our Director of Community Engagement, Sarah Garwood at sg@rtwf.org.

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Annual Summit Registration Link is LIVE!

COMBINEDBrain — Thu, 18 Jun 2026 21:21:54 GMT

Please click this link to register for the Annual Summit to be held on November 2-3, 2026 in Nashville, TN! PAG members, clinicians, researchers, and industry partners are welcome. Like last year, we will take one registration per PAG and others can be added to the waiting list if there is space available.

International Conference on Clinical Research Networks for Rare Diseases

COMBINEDBrain — Thu, 11 Jun 2026 14:43:34 GMT

The IRDiRC is pleased to announce that the meeting report from the 2^nd International Conference on Clinical Research Networks for Rare Diseases is now available. This report has been jointly developed by ERDERA, Rare Diseases International, and IRDiRC.

Held on 9-10 December 2025 in Heidelberg, Germany, in a hybrid format, the conference brought together global experts and stakeholders under the theme “Mobilizing the Global Rare Disease Clinical Research Ecosystem”. Building on the success of the inaugural edition in 2022, the event convened researchers, clinicians, patient advocates, industry representatives, policymakers and funders to strengthen international collaboration in rare disease clinical research.

Report: https://www.rarediseasesinternational.org/wp-content/uploads/2026/06/CRN-Conference-Final-Report.pdf

Clinical Research Networks play a crucial role in accelerating progress rare diseases by connecting expert centers, harmonizing protocols, and enabling data sharing across borders. The insights captured in the report aim to support continued collaboration and provide actionable pathways to strengthen and sustain these networks globally.

We kindly invite you to support the dissemination of this report within your networks and communities.

IRDiRC Publication

COMBINEDBrain — Mon, 01 Jun 2026 14:16:40 GMT

We are pleased to share that a new IRDiRC publication, “Rare disease preparedness: the time is now” has been recently published in the Rare Disease and Orphan Drugs Journal. The paper calls for urgent, coordinated global action to transform how rare diseases are diagnosed, treated, and prioritized within the healthcare systems. The publication is open access.

Read the full publication: https://www.oaepublish.com/articles/rdodj.2025.39?utm_campaign=website&utm_medium=email&utm_source=sendgrid.com

We kindly ask you to help us with the dissemination of this publication and sharing it with your networks.

Changes to Roadshow Schedule

COMBINEDBrain — Wed, 27 May 2026 20:33:38 GMT

Please note, the following roadshow has been canceled:

GLUT1 - July 14-15, 2026 - Asheville, NC

As a reminder, there is another opportunity to donate in North Carolina, a 2.5hr drive away in Concord, NC on June 25-26.

And, there are two additional roadshow locations:

CureVCP - July 23-24, 2026 - Atlanta, GA - TBD

Rory Belle Foundation - August 8, 2026 - Boston, MA - TBD

Please contact biorepository@combinedbrain.org with questions.

Biorepository Roadshows Update

COMBINEDBrain — Wed, 06 May 2026 19:37:19 GMT

If you haven't been to a COMBINEDBrain Roadshow yet, here's what you're missing!

There is still room for non-hosting PAGs to send donors for sample collections at some of our locations. Please contact biorepository@combinedbrain.org for more information.

Annual Summit Dates and Location

COMBINEDBrain — Fri, 27 Mar 2026 19:06:16 GMT

Mark your calendar!

The 2026 COMBINEDBrain Annual Summit will be held on November 2-3, 2026. We will be at the Hilton Garden Inn Nashville Vanderbilt, 1715 Broadway, Nashville, TN 38203. We'll hold our general meeting on November 2 and offer a Drug Repurposing Bootcamp on November 3.

The event registration page will be coming soon ... watch this news page for more details.

HOPE TO SEE YOU THERE!