Rare Disease Legislative Advocates

09/25/2025

Happening today! Join us at 3:00 PM ET for the latest RDLA webinar. This webinar features four insightful speakers and will focus on rare disease legislative updates and how advocates can support.

Register for the event here➡️ https://hubs.li/Q03K-HHN0

09/22/2025

We are excited for Rare Disease Week 2026 on Capitol Hill this coming February. To help participants attend, we offer a limited number of travel reimbursements. The application is open now! The application window closes on Friday, November 7.

There are also exciting programming improvements coming to Rare Disease Week 2026 based on community feedback. To learn about all the improvements and apply for a travel reimbursement, click here: https://hubs.li/Q03Kj5ZY0.

09/11/2025

Today, RDLA hosted the latest Rare Disease Congressional Caucus Briefing. The briefing focused on regulatory urgency and therapeutic opportunity in the drug approval process. Our amazing guest speakers came from different areas of the rare disease community to share their testimony. Here's what they had to say:

"The rare disease community has been advocating for greater predictability, consistency, and transparency." - James Valentine, an FDA lawyer and Director at Hyman, Phelps & McNamara, P.C.

"We must advance regulatory consideration and patient testimonies to be beyond anecdotes, but rather to be included as critical data in the compressive and thorough review of new drug applications." - Emily Milligan, Executive Director at the Barth Syndrome Foundation

"Delayed approvals cost the government more money. We need Congress to ensure the FDA has resources, incentives, and structures to serve our children. " - Jessica Haywood, a board member at the Cure Sanfilippo Foundation

"As programs are being paused muscles are wasted and families are losing hope. With your action, Congress can make a difference." - Kelly Brazzo, the Co-Founder/CEO of the Curelgmd2i Foundation

"Our rare disease community wants, needs, and deserves safe and effective therapies as quickly as possible. We don't have time to wait; we need therapies for this generation of patients." - Annie Kennedy, the Chief of Policy, Advocacy, and Patient Engagement at the EveryLife Foundation for Rare Diseases

We want to express our gratitude to these patient advocacy champions for their heartfelt testimonies and stories.

In case you missed it earlier today, you can watch the full briefing on YouTube ➡️ https://youtube.com/live/touiS3iV-yk

09/11/2025

Join RDLA and the Rare Disease Congressional Caucus today at 2 PM ET for a vital conversation focused on regulatory urgency and therapeutic opportunity.

The event will take place in House Rayburn 2060, Washington, D.C., and via livestream

Register now! https://hubs.ly/Q03H_78B0

09/02/2025

September is Newborn Screening Awareness Month, and we have a full calendar of opportunities for you to learn and engage!

📅 Sep. 12: Newborn Screening Evidence Review Workshop – Presentation and discussion of completed evidence reviews for Duchenne and MLD.

📅 Sep. 17: Newborn Screening Bootcamp – A virtual training co-hosted with Expecting Health for advocates to engage with the newborn screening system.

📅 Sep. 25: RDLA Community Webinar – Stay up to date on the latest rare disease policy developments.

Join us this Newborn Screening Awareness Month to learn more about what is being done and how you can help protect and strengthen this life-saving program

👉 To register and find more details visit our events calendar here: https://everylifefoundation.org/events-schedule/

08/27/2025

Attention Lansing, Michigan!

RDLA, in collaboration with the Michigan Rare Coalition, is hosting a day of action for Michigan residents impacted by rare disease to join together and meet your state legislators, share your stories, and help advance the policy priorities of the rare disease community.

Who: This event is open to Michigan residents only. No prior advocacy experience is necessary. Registration for this event and all RDLA events are free for all rare disease advocates. Registration is required for all participants, including spouses and children.

When: September 30 from 8 AM to 5 PM EST.

Register Here: https://share.hsforms.com/1eaFAXt8IQYOXxHRuTjYfZQdj7j2

08/19/2025

Today, on National Patient Advocacy Day, we honor and celebrate the relentless advocates who give voice to the 30 million Americans living with rare diseases. Your courage, determination, and persistence continue to drive meaningful change and bring hope to countless families.
💜 Know an advocate who’s made a difference in your life? Tag them! Or take it a step further and make a gift to the EveryLife Foundation in their honor, and we’ll send them a personal thank-you note on your behalf. Let's thank those that inspire us! https://everylifefoundation.org/donate/

08/18/2025

The EveryLife Foundation for Rare Diseases is heartbroken to learn of the passing of Jacob Venditti. Jacob was an invaluable member of our RDLA Advisory Committee. Since 2024, his optimistic and kindhearted dedication to the rare disease community ensured rare disease patients like him have a voice in making change.

Jacob was the founder of Live Fearlessly Foundation, a nonprofit organization that empowers young people with cystic fibrosis (CF) to pursue extracurricular athletic activities of their choice. We are grateful to have witnessed firsthand his ability to live fearlessly himself, and we will continue to be inspired by his relentless passion for the rare disease community.

To learn more about Jacob, visit https://livefearlesslyfoundation.com/

In the photo below, Jacob is pictured next to EveryLife Foundation CEO Michael Pearlmutter at the 2024 RareVoice Awards.

08/18/2025

Thank you to every advocate who made Rare Across America 2025 unforgettable! You took our message straight to Congress, sharing your stories, building relationships, and pushing for policies that matter to the rare disease community.

Let's keep the momentum going—share your own Rare Across America moments using !

08/13/2025

The Federal Advocacy – Congressional Staff Award honors congressional staffers who have gone above and beyond to create and enact policies that improve the lives of those affected by rare diseases.

These behind-the-scenes champions work tirelessly to shape legislation, support constituents, and drive change that matters. Let’s make sure their work gets the recognition it deserves.

📝 Nominate them today: https://hubs.li/Q03CDBn40

07/30/2025

Happening TOMORROW, Thursday, July 31 at 3pm ET! Join EveryLife for our July State Advocacy webinar to discuss coalition-building in the rare disease space.

This webinar will highlight how organizations are partnering effectively to drive real policy change within and across states. Whether you’re part of a nonprofit, a patient group, or a solo advocate, there’s something here for you!

🔗 Register here: https://hubs.li/Q03zBT3m0

07/29/2025

Nominations are now open for the 2025 RareVoice Awards! Help us honor advocates who amplify the voice of the rare disease community and drive change in state and federal policy on Capitol Hill.

✅ Speak out for change
✅ Push policy forward
✅ Empower the rare disease community

If someone comes to mind, take just 5 minutes to nominate them today: https://shorturl.at/lTm83

Please note that individuals and organizations may be a finalist nominee two years in a row, but may not be nominated again until two years have passed since the last nomination. In addition, a former awardee of a RareVoice Award will not be eligible for another nomination for three years.