Our Patient Advocacy Pavilion had exhibitors from 20+ patient advocacy organizations.

Pamela Gavin, CEO of NORD, giving the opening remarks at the NORD Breakthrough Summit.

Maria Kefalas, Founder of the Calliope Joy Foundation and Cure MLD gives a powerful keynote speech at the Summit: “Some of the most amazing advocacy comes from bringing meals to a family in the hospital or just giving testimony to the FDA. You don’t need to save everyone. It is more than enough to do something, anything.”

Tanita Allen, a health equity advocate with Huntington’s Disease, speaking in front of conference attendees about racial biases experienced in her diagnostic odyssey.

Maria Miller, a fourth-year medical student living with a rare disease, speaking to the crowd during the opening keynote address.

Dr. Joni Rutter, Director of the National Center for Advancing Translational Sciences (NCATS), NIH

The Patient Advocacy Pavilion housed many informative discussions as seen here between one attendee and an Uplifting Athletes representee.

Attendee speaking with NORD's Vice President of Patient Services Jill Pollander learning about all that NORD does in the rare disease space.

From left to right: NORD Vice President of Community and Corporate Affairs, Patrick Collins; Rachid Izzar of Biogen; Julie Kim of Takeda; Kate Haviland of Blueprint Medicines; Giacomo Chiesi of Chiesi Global Rare Diseases; Brian Goff of Agios Pharmaceuticals

From left to right: NORD Vice President of Community and Corporate Affairs, Patrick Collins; Rachid Izzar of Biogen; Julie Kim of Takeda; Kate Haviland of Blueprint Medicines; Giacomo Chiesi of Chiesi Global Rare Diseases; Brian Goff of Agios Pharmaceuticals

From left to right: Panelists from our ”Embracing Innovation to Enhance Clinical Trial Success” session included Dr. Rachele Hendricks-Sturrup, DHSc, MSc, MA, Real-World Evidence (RWE), Duke-Margolis Institute for Health Policy; Patrizia Cavazzoni, MD, Center for Drug Evaluation & Research (CDER); U.S. Food & Drug Administration P.J. Brooks, PhD, NCATS, NIH; Mat Davis, Jazz Pharmaceuticals; and Maria Santaella, PhD(c), MSN, RN-BC, CPHON, National Bleeding Disorders Foundation.

We are grateful for our dedicated sponsors like Moderna.

Dr. Joel Ross shaking hands with Giacomo Chiesi of Chiesi Global Rare Diseases.

From left to right: NORD Vice President of Community and Corporate Affairs, Patrick Collins; Rachid Izzar of Biogen; Julie Kim of Takeda; Kate Haviland of Blueprint Medicines; Giacomo Chiesi of Chiesi Global Rare Diseases; Brian Goff of Agios Pharmaceuticals; and NORD CEO Pamela Gavin

Attendees were eager to ask questions during Q&A in one of the breakout sessions on day one of the Summit.

From left to right: NORD Vice President of Community and Corporate Affairs, Patrick Collins; Rachid Izzar of Biogen; Julie Kim of Takeda; Kate Haviland of Blueprint Medicines; Giacomo Chiesi of Chiesi Global Rare Diseases; Brian Goff of Agios Pharmaceuticals

"Can We Make Gene Therapy More Accessible?" panel. From left to right: P.J. Brooks, PhD, Division of Rare Diseases Research Innovation, NCATS, NIH; Peter Marks, MD, PhD, , CBER, FDA; Nicole Gaudelli, GB; David Barrett, JD, American Society for Gene and Cell Therapy (ASGCT)

Jeneva Stone, esteemed poet and Maryland Rare Action Network® Ambassador, and her son, Rob.

"The Growing Role of Medical Devices" panel. Moderating the panel is Joseph Sapiente, Medical Device Innovation Consortium (MDIC). Sitting to his right is Michelle Tarver, MD, PhD, Center for Devices and Radiological Health, FDA; Matt Wetzel, Goodwin Procter LLP; Rich Brennan, ALS Association

Our platinum sponsor, Sanofi was onsite to talk about the work they do in the rare disease space.

Balancing Cost, Innovation and Access panel. Moderator: Heidi Ross, NORD. Seated to her left is Mary Dwight, Cystic Fibrosis Foundation | Lee Fleisher, MD, Rubrum Advising

Balancing Cost, Innovation and Access panel. Moderator: Heidi Ross, MPH, NORD. Seated to her left is Mary Dwight, Cystic Fibrosis Foundation | Lee Fleisher, MD, LLM, Rubrum Advising | Gemma Mayman, Pfizer | R. Duane Clark, Sanofi | Jason Spangler MD, MPH, Center for Innovation & Value Research

Conversation flowed between attendees and sponsors like Takeda.

There was ample opportunity for 1:1 networking with industry peers to foster collaboration and innovation.

"The Growing Role of Medical Devices" panel. Moderating the panel is Joseph Sapiente, Medical Device Innovation Consortium (MDIC). Sitting to his right is Michelle Tarver, MD, PhD, CDRH, FDA | Matt Wetzel, Goodwin Procter LLP | Rich Brennan, M.A., ALS Association

Pfizer came prepared to educate and inform how they are contributing to progress in the rare disease space.

Attendees had the chance to learn about the work of NORD's sponsors and partners at the exhibit hall like Medlive, our exclusive rare disease Continuing Medical Education (CME) partner.

Lightning Round poster presenter, Kelly McNeil-Posey, RN, MBA, Alexion, AstraZeneca Rare Disease, presents their research: Developing new diversity measures to ensure fair representation in clinical trials.

Lightning Round poster presenter Kelly McNeil-Posey, RN, MBA, Alexion, AstraZeneca Rare Disease presents her poster: "Developing new diversity measures to ensure fair representation in clinical trials."

Lightning Round poster presenter, Aleksandra Foksinka, MS, University of Alabama at Birmingham presents their poster: SUmmer in PREcision MEdicine (SUPREME): An Immersive Research Experience in Precision and Genomic Medicine.

Lightning Round poster presenter, Geetanjoli Banerjee, PhD, MPH, Moderna, presents her poster: The Clinical Burden of Propionic Acidemia in Patients with or without Metabolic Decompensation Events Stratified by Age, in the United States.

Lightning Round poster presenter Vladislav Kovalik, MD, MSPH, Mercy Medical Center presents his poster: Empowering Patient-Funded Research for Rare Cancers at a Community Hospital: A Success Story in Peritoneal Surface Malignancy.

Our Poster Hall featured 75+ posters about the latest rare disease research

Aleksandra Foksinka, MS, University of Alabama at Birmingham, discusses their research poster, "SUmmer in PREcision MEdicine (SUPREME): An Immersive Research Experience in Precision and Genomic Medicine" which was selected for lightening round presentation.

Patients and caregivers are both effective advocates for people living with rare diseases!

Attendees showed their love of NORD's mascot, NORDY!

No NORD event would be complete without NORDY the Zebra!

The exhibit hall allowed for new connections to be formed, strengthening the rare disease industry.

The poster hall presented a plethora of opportunities to learn and network.

Patient-driven study design and implementation in an app-based research program measuring home-reported outcomes (HROs) in paroxysmal nocturnal hemoglobinuria (PNH). Pictured from left to right are Glorian Yen (Novartis), Lincy Geevarghese (Novartis), Samantha McStocker (Folia Health), and Amanda Healey (Folia Health). Lincy was not the author of the poster pictured.

Pamela Gavin, NORD CEO (left) posed with Dr. Robert Califf, FDA Commissioner (middle) and Dr. Meena Seshamani, CMS Deputy Administrator and Director of the Center for Medicare (right)

Panel: Bringing Research to Patients to Be More Inclusive. Speakers included: Joni L. Rutter, Ph. D, NCATS, NIH | Kristin Schneeman, FasterCures, Milken Institute Health | Jim Palma, TargetCancer Foundation | Jenifer Waldrop, MS, Rare Disease Diversity Coalition | Moderator: Edward Neilan, MD, PhD, NORD

Panel: Bringing Research to Patients to Be More Inclusive. Speakers included: Joni L. Rutter, Ph. D, NCATS, NIH | Kristin Schneeman, FasterCures, Milken Institute Health | Jim Palma, TargetCancer Foundation | Jenifer Waldrop, MS, Rare Disease Diversity Coalition | Moderator: Edward Neilan, MD, PhD, NORD

Mousumi Bose, Ph. D, Department of Nutrition and Food Studies, College for Community Health, Montclair State University delivers the Patient/Caregiver keynote: The Roar of Rare: How Advocating for My Child's Care Helped Me Find My Voice in Research.

Pamela Gavin, NORD CEO (middle) posed with Dr. Robert Califf, FDA Commissioner (left) and Dr. Meena Seshamani, CMS Deputy Administrator and Director of the Center for Medicare (right).

Pamela Gavin, NORD (left) posed with Dr. Robert Califf, FDA (middle) and Dr. Meena Seshamani, CMS, Center for Medicare (right) held a fireside chat entitled: From Drug Approval to Patient Access.

Brian Connell, Leukemia & Lymphoma Society; Jennifer Dexter, National Health Council; Jordan Brossi, Office of Congresswoman Anna G. Eshoo (CA-16); Barrett Tenbarge, Ranking Member Cassidy, Senate Health, Education, Labor, and Pensions (HELP) Committee and Heidi Ross, MPH, NORD discuss Policy Priorities in Rare for 2025 and Beyond.

Jennifer Dexter, National Health Council; Jordan Brossi, Office of Congresswoman Anna G. Eshoo (CA-16); Barrett Tenbarge, Ranking Member Cassidy, Senate Health, Education, Labor, and Pensions (HELP) Committee and Heidi Ross, MPH, NORD discuss Policy Priorities in Rare for 2025 and Beyond.

Seated next to Gabrielle Rushing, PhD, CSNK2A1 Foundation is Yasmine Wasfi, MD, PhD, Savara, who is speaking to the audience.

Martin Mackay, PhD, RallyBio speaks to the audience at the panel: "How Investors see the Rare Space."

How Investors See the Rare Space Now answers the question of an audience member. Seated from Left to right is moderator, David Scheer, Scheer & Company, Inc. | Tal Zaks, MD, PhD, OrbiMed | Maha Radhakrishnan, M.D, Sofinnova Investments | Martin Mackay, PhD, RallyBio | Sukumar Nagendran, MD, Taysha Gene Therapies

Kristen Wheeden, MBA, President, United Porphyrias Association delivers an opening keynote on behalf of Mayra Martinez.

Panel: How Can AI Help the Rare Community NOW? On stage from left to right is: Nara Sobreira, MD, PhD, McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University | Qi Liu, PhD, MStat, FCP, OCP, OTS, CDER, FDA | Joseph Zabinski, PhD, MEM, OM1 | Moderator: Greg Levine, JD, Ropes & Gray LLP

The Current State & Outlook for Newborn Screening session. (l-r) Joel Cartner, Esq., Muscular Dystrophy Association (MDA) | Mei Baker, MD, FACMG, University of Wisconsin School of Medicine and Public Health; Wisconsin State Laboratory of Hygiene | Holly Peay, PhD, MS, RTI International

Guadalupe Hayes-Mota, MBA, MS, MPA, CEO, Healr Solutions, and Steering Committee Member of the Massachusetts RDAC provided a powerful keynote on Day Two.

Sal J Morana RPh, PhD, EVP, Alliant Employee Benefits speaks to the audience at the "Navigating the Coverage Hurdles to Rare Disease Access" panel.

Panel: Navigating the Coverage Hurdles to Rare Disease Access. Seated (from left to right) is moderator: Michelle Rice, Michelle Rice and Associates | Kellyn Madden, MS, Friedreich’s Ataxia Research Alliance (FARA) | Lauren Walrath, Kyowa Kirin, Inc. | Sal J Morana RPh, PhD, EVP, Alliant Employee Benefits | Mike Baldzicki, CRCM, Lyceum

Smiles were in abundance at the 2024 Summit!

Attendees listen to the panel "Enhancing the Power of Patient Data." Seated from left to right is the moderator, Gabrielle Rushing, PhD, CSNK2A1 Foundation | Yasmine Wasfi, MD, PhD, Savara | Rosa Sherafat-Kazemzadeh, MD, Office of Clinical Evaluation | Jill Kiernan, TBRS Community | Kimberly Smith, MD, MS, Office of Medical Policy, CDER, FDA