Honoring pioneers in rare disease research, advocates spearheading change, and innovative companies whose treatments have significantly improved rare disease patient outcomes.

About

The National Organization for Rare Disorders (NORD®) is proud to honor the achievements of our community. Each year, we recognize rare disease heroes —individuals or groups who are driving progress and making a difference on behalf of the more than 30 million Americans living with rare diseases.

Rare Impact Awards® categories are as follows:

  • Policy Changemaker
    Recognizes federal and state policymakers who have advanced policies or regulations that improve the lives of those living with rare disease in the United States.
  • Community Champion
    Honors patient advocates who, individually or as part of a group, have made a meaningful impact in helping to achieve NORD’s mission by improving access to care, accelerating research, or driving policy.
  • Youth Leader
    Celebrates individuals under the age of 18 who have made an outstanding contribution by volunteering their time and efforts to raise awareness of rare diseases.
  • Scientific and Medical Trailblazer
    Recognizes individuals who have made significant contributions to the rare disease community through clinical care or research.
  • Abbey S. Meyers Leadership Award
    Honors a NORD patient advocacy organization member that has made significant contributions to the rare disease community through strong leadership in advocacy, education, research, patient support services, or raising awareness.

Meet the 2025 Honorees

The 2025 Rare Impact Award Honorees are an extraordinary and diverse group that includes pioneers in genetic research, longstanding patient advocacy leaders, and innovative companies whose FDA-approved treatments have significantly improved rare disease patient outcomes. Each Honoree’s story is a testament to the power of rare innovation and perseverance.

Community Champion

Robert Graham of New York

Bob Graham exemplifies the definition of a Community Champion through his multifaceted contributions to New York’s rare disease community.

Community Champion

Jaime McHugh of New Hampshire

Jaime has had a tremendous impact in helping to achieve NORD’s mission, by driving policy, spreading awareness for the entire RARE disease community, and fundraising by running her second Boston marathon as a second time Running4Rare Champion.

Community Champion

Mary Nadon Scott of Vermont

As a founding member of the NORD Vermont Rare Disease Advisory Council Coalition, Mary has been instrumental in building a strong, inclusive community that advocates for all those affected by rare diseases.

Medical & Scientific Trailblazer

Angela Scheuerle, MD of Texas

Dr. Scheuerle is the Director of the NORD Rare Disease Centers of Excellence at the University of Texas-Southwestern.

Medical & Scientific Trailblazer

Jerry Vockley, MD, PhD of Pennsylvania

Dr. Vockley is the Co-Director of the NORD Rare Disease Center of Excellence at the University of Pittsburg and UPMC Children’s Hospital of Pittsburgh, where he has dedicated his career to advancing rare disease research and care.

Policy Changemaker

The Honorable Tammy Baldwin of Wisconsin

U.S. Senator Tammy Baldwin has established herself as a steadfast champion for the rare disease community through her consistent legislative leadership and bipartisan collaboration on critical healthcare initiatives.

Policy Changemaker

The Honorable Kevin Blackwell of Mississippi

State Senator Kevin Blackwell sponsored the Mississippi Rare Disease Advisory Council (RDAC) legislation and played a pivotal role in establishing this critical body that gives voice to the rare disease community in Mississippi.

The Abbey S. Meyers Leadership Award

Hermansky-Pudlak Syndrome Network

Led by Executive Director and Founder, Donna Appell and her daughter Ashley, who lives with HPS, Hermansky-Pudlak Syndrome Network exemplifies extraordinary leadership in rare disease advocacy.

Industry Honorees

Industry Innovation Award

argenx for VYVGART Hytrulo®
Industry Innovation Award

Ascendis Pharma for YORVIPATH®
Industry Innovation Award

BridgeBio for Attruby™
Industry Innovation Award

Ionis Pharmaceuticals for TRYNGOLZA™
Industry Innovation Award

Orchard Therapeutics for LENMELDY™
Industry Innovation Award

PTC Therapueutics for KEBILIDI™
Industry Innovation Award

X4 Pharmaceuticals for XOLREMDI®
View All

Frequently Asked Questions

The Rare Impact Awards represent our annual celebration of individuals, organizations, and companies working to better the lives of the rare disease community through advocacy, research, and drug development.

We’ve taken an opportunity to reflect on how we may better celebrate the extraordinary achievements in rare disease research, innovation, and advocacy with more members of the rare disease community. As a result, our 2025 honorees will be recognized throughout the year in various ways, including expanded reception-style celebrations at our largest events. In this new format, honorees will benefit from broader visibility and recognition among peers and colleagues in the rare disease community.
  • Scientific and Medical Trailblazer Awards will be recognized at the NORD® Rare Disease Scientific Symposium on June 2-3 in Washington, D.C.
  • Community Champion, Policy Changemaker, Youth Leader, and Abbey S. Meyers Leadership Awards will be celebrated during our annual NORD® Rare Diseases and Orphan Products Breakthrough Summit on October 19-21 in Washington, D.C.
  • Industry Innovation Awards will be presented to each recipient at their U.S. headquarters, where recognition will be shared with employees across various functions at the organization.

For more information about NORD’s Rare Impact Awards and the NORD events where honorees will be recognized, please contact: [email protected]

Thank You for Helping Us Celebrate the 2024 Stars of Rare Disease Advocacy and Innovation

Thank you for joining us at the 2024 Rare Impact Awards live from Universal Studios, Hollywood. If you were unable to attend the event, you can check out our favorite highlights from this unforgettable night and explore each of our 2024 honorees below.

You can also watch the entire ceremony on YouTube, here.