From Voices to Breakthroughs

OCTOBER 19-21, 2025

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ATTENDEES

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SPEAKERS

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SPONSORS AND EXHIBITORS

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RESEARCH POSTERS

Shape the future of rare disease treatments, research and policy at the NORD® Rare Diseases and Orphan Products Breakthrough Summit.® Together, we can advance innovation for the more than 30 million Americans – and more than 400 million people worldwide – with rare diseases!

Why Attend?

Collaborate & Influence

Contribute to critical discussions, forge valuable partnerships, and help advance equitable access to rare disease research and
patient care.

Engage with Experts

Connect with influential voices and stakeholders shaping the future of rare disease treatments, research and policy.

Discover Innovations

Learn about cutting-edge advancements to improve access to clinical trials, and discover emerging trends and issues that are poised to redefine the landscape of rare disease.

Some of the rare disease leaders and innovators you’ll hear from at the 2025 NORD Breakthrough Summit

David Scheer

President

Scheer & Company

Paula Ragan, PhD

Chief Executive Officer and President

X4 Pharmaceuticals

Miguel Fernandez Alcalde

President

EMD Serono

Catherine Owen Adams, BSc.

Chief Executive Officer

Acadia Pharmaceuticals

Bruce Bloom

Chief Science Officer

Kabuki Syndrome Foundation

Maha Radhakrishnan, MD

Executive Partner

Sofinnova Investments

Sean Nolan

Chief Executive Officer

Taysha Gene Therapies

Theresa Strong

Director of Research Programs

Foundation for Prader-Willi Research

See Full Speaker List

Featured Sponsors

Platinum Sponsor

Gold Sponsor

View All Sponsors Learn About Sponsorship Opportunities

What Past Attendees Had to Say

“A world-class event that brings together researchers, pharmaceutical companies, medical professionals, and families affected by rare disorders. Nearly 1,000 attendees, each with a unique story and perspective on living with or supporting someone with a rare disorder. The atmosphere was electric with a sense of purpose and unity.”

- Rare Community Member

“Attending such a prestigious event as a student was truly invaluable, both professionally and personally. I gained so much from the presentations, had insightful conversations with experts in the rare disease community, and left feeling more inspired than ever to contribute to this field.”

- M.S. Candidate

“The NORD Breakthrough Summit provided an incredible platform for sharing stories, for bringing attention to the unmet needs of the rare disease community, and for pushing forward the conversation around better care, research, and advocacy.”

- Patient Advocacy Organization Member

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INDEPENDENCE IN EDUCATIONAL CONTENT AT NORD CONFERENCES AND MEETINGS

NORD is committed to ensuring that the educational content presented at our conferences and meetings is developed with integrity, objectivity, and independence. Our educational session topics and faculty are planned and selected without influence from industry partners, sponsors, or any external entities that may have a commercial interest in the topics discussed.

Content is determined solely by NORD staff, planning committees, and subject matter experts.

NORD speaking opportunities are never granted in exchange for sponsorship, membership, financial contributions, or any other paid affiliation. All speakers must disclose any financial or professional relationships that may pose a conflict of interest. Speaker panels are composed to reflect varied perspectives, ensuring balanced discussions free from undue influence.