News

THANK YOU TO OUR GENEROUS ANNUAL SUMMIT SPONSORS!

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

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!

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!

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!

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

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. LinkedIn Instagram Facebook YouTube Copyright © 2026 RTW Foundation, All rights reserved. You are receiving this email because you opted in via our website. Our mailing address is: RTW Foundation 40 10th Ave Fl 3 New York, NY 10014-1115 Add us to your address book Want to change how you receive these emails? You can update your preferences or unsubscribe from this list.

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.

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.

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. 

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.