Global Genes

Global Genes Empowering the Next Generation Rare Disease Advocate. Merged with RARE-X Dec. 2022. Hope. It’s in our genes.™

The early bird gets the savings… but only until June 26th!We are officially counting down to the RARE Drug Development S...
06/23/2026

The early bird gets the savings… but only until June 26th!

We are officially counting down to the RARE Drug Development Symposium this September in Boston! This year’s event is entirely built around a powerful mission: "Accelerating Rare Disease Progress: Aligning Advocates, Science & Industry."

Breakthroughs don't happen in silos. True to our sub-theme of Advocates Powering Research, this symposium is where patient advocates, industry innovators, and top researchers align to challenge ideas, collaborate, and speed up the therapeutic pipeline.

Don't wait until the clock runs out on Friday, June 26th and the price jumps. Secure your registration at the lowest possible rate today!

Head to the link in our bio and click REGISTER NOW!
For full event details, visit globalgenes.org

This Father's Day, we're honoring every rare disease dad who shows up at appointments, in waiting rooms, in the middle o...
06/21/2026

This Father's Day, we're honoring every rare disease dad who shows up at appointments, in waiting rooms, in the middle of the night, and in every moment in between. 💙

To the dads who became experts overnight, who fought for diagnoses, who held their families together through the hardest chapters, you are seen and you are celebrated!

Being a rare disease dad means loving fiercely in the face of uncertainty. It means advocating loudly for someone who means everything. It means never giving up.

Happy Father's Day to all the dads in our rare disease community. You make a difference every single day! 💙

The FDA’s playbook for rare disease drug development is evolving, and you need to be in the room. 🏛️✨For Session 4 of RD...
06/17/2026

The FDA’s playbook for rare disease drug development is evolving, and you need to be in the room. 🏛️✨

For Session 4 of RDDS, we’re diving deep into Regulatory Update: Innovative Paths to Therapy Development and Scalability.

We are thrilled to host Amy Comstock Rick, J.D. (CDER's Associate Director for Rare Disease Strategy & Director of Strategic Coalitions for the FDA's Rare Disease Innovation Hub) for an exclusive look at how the agency is streamlining the approval pipeline.

Amy will be covering:
🔹 The Innovation Hub’s strategic agenda
🔹 Understanding the “Plausible Mechanisms Framework”
🔹 Designing trials with novel endpoints
🔹 How advocacy groups can champion change directly with the FDA

Whether you’re a researcher, developer, or patient advocate, this session will give you the roadmap you need to accelerate life-changing treatments.

🔗 Click the link in our bio to view the full agenda and grab your ticket!

From a single gene discovery to a real-world treatment on the shelf - the drug development journey is a massive team spo...
06/11/2026

From a single gene discovery to a real-world treatment on the shelf - the drug development journey is a massive team sport.

If you’ve ever wondered how patient communities successfully navigate the long road through preclinical work, clinical trials, and FDA approval, our next RDDS featured session is for you!

We’re doing a Therapeutic Timeline Deep Dive to look at the exact handoffs, setbacks, and synergies that matter most. To bring this to life, we’re spotlighting two incredible communities leading the charge:

🧬 Angelman Syndrome: Through powerful advocacy/industry partnerships, they now have multiple “Breakthrough Therapy”-designated candidates in active clinical trials.
💊 Niemann-Pick Type C (NPC): Celebrating the recent FDA approval of TWO treatments, with a third anticipated this year!

Whether you’re a patient, advocate, researcher, or industry partner, come learn the real-world blueprint to accelerating therapeutic research.

🗓️ Tap the link in our bio to reserve your spot!

Rare. Proud. Unstoppable. 🏳️‍🌈✨This June, we celebrate the intersection of two communities defined by strength, resilien...
06/03/2026

Rare. Proud. Unstoppable. 🏳️‍🌈✨

This June, we celebrate the intersection of two communities defined by strength, resilience, and the courage to be seen.

Happy Pride Month to every LGBTQA+ patient, caregiver, researcher, and advocate in the rare disease world. You make this community brighter. 🌈

🎧 RARECast is the weekly Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the inters...
05/11/2026

🎧 RARECast is the weekly Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare diseases with business, science, and policy, and is supported by the members of the Global Genes Corporate Alliance.

April Episodes Included:
⭐ Špela Miroševič, Researcher, Co-Founder & President, CTNNB1 Foundation
⭐ Global Head, Rare and Neurologic Diseases Therapeutic Area at Sanofi, Pablo Sardi
⭐ Jim Foote, Co-founder and CEO, First Ascent Biomedical
⭐ Nicole Johnson, Executive Director of FOXG1 Research Foundation and Nasha Fitter, CEO of FOXG1 Research Foundation
⭐ Co-Founder, Chief Medical & Scientific Officer, MyOme, Akash Kumar

Listen to RARECast Here: https://globalgenes.org/podcasts/

Happy Mother's Day to the caregivers who mother beyond measure💙
05/10/2026

Happy Mother's Day to the caregivers who mother beyond measure💙

We are thrilled to announce that registration is OPEN for the 2026 RARE Drug Development Symposium! 🎉 This premier event...
05/06/2026

We are thrilled to announce that registration is OPEN for the 2026 RARE Drug Development Symposium! 🎉 This premier event, hosted by Global Genes, is a critical gathering for anyone passionate about driving progress in rare disease treatments.

Join us from September 9-11, 2026, in the beautiful city of Boston, MA. Get ready for a dynamic lineup of speakers, workshops, and networking opportunities. It's the perfect environment for patient advocates, researchers, and industry leaders to come together and collaborate.

Spots fill up fast, so click the link in our bio to register today! 👇
https://globalgenes.org/rdds-2026/
We can't wait to see you there!

We want to see YOU at the 2026 RARE Drug Development Symposium! 🗣️💡Financial barriers shouldn't stand in the way of advo...
04/22/2026

We want to see YOU at the 2026 RARE Drug Development Symposium! 🗣️💡

Financial barriers shouldn't stand in the way of advocacy. We are thrilled to announce that applications for the Advocate Support Travel Stipend are officially OPEN!

This program is designed to help offset meeting and travel expenses, making it easier for our community leaders to join the conversation on rare disease innovation.

Who is eligible to apply?

🧬 Patients & Advocates: Individual patients and dedicated rare disease advocates.
👨‍👩‍👧‍👦 Support Systems: Close family and friends of advocates.
🤝 Organization Leaders: Paid or volunteer staff of rare disease nonprofits and support groups.
Your voice is essential to the future of drug development. Apply now to secure support for your journey!

https://globalgenes.tfaforms.net/f/rdds2026-advocatesupport

Address

1012 14th Street NW Suite 500
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20005

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Tuesday 8am - 5pm
Wednesday 8am - 5pm
Thursday 8am - 5pm
Friday 8am - 5pm

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