National Organization for Rare Disorders, Inc. (NORD)

08/22/2026

You can make a difference for the community! The Global PNH Patient Registry is now seeking participants interested in moving research forward — powered by patients. Sign-up now: pnh.iamrare.org

08/22/2026

Looking for a meaningful way to support the 1 in 10 Americans living with a rare disease?

RSVP to join us on Aug. 27 from 4–5 p.m. ET for a virtual information session to learn about volunteering with NORD: https://bit.ly/3LXYi3d

Whether you’re interested in engaging with lawmakers, running for rare, or sharing your story, there’s a place for you on our team.

08/21/2026

Cheers to Maeve and an unforgettable night of FUNdraising! 🏃‍♀️🍻

Our 2026 NORD Running for Rare runner Maeve stepped behind the bar at Eastpoint Bar for a special happy hour supporting her journey to the 2026 TCS New York City Marathon!

A portion of the evening’s proceeds supported Maeve’s fundraising efforts for NORD. Maeve’s Rare Patient Community Partner, fellow runners, and NORD team member Jaime joined the celebration!

Thank you, Maeve and Eastpoint Bar, for helping us raise awareness and support for the rare disease community.

New York Road Runners (NYRR)

08/21/2026

We’re shining a light on DYRK1A syndrome for 🧡 This rare genetic condition can shape how a person develops, learns, communicates, eats, and grows.

NORD Member Organization Dyrk1a Syndrome International Association helps families facing this disorder connect, find resources, and support research efforts. Learn more at dyrk1a.org.

08/21/2026

NORD was proud to present Jazz Pharmaceuticals with a 2026 Industry Innovation for developing the first and only FDA-approved treatment for one of the most aggressive brain tumors impacting children and young adults, recurrent H3K27M-mutant diffuse midline glioma (DMG).

Every year, about 2,000 Americans and their families receive this devastating rare cancer diagnosis. Their community spent more than a decade, through organizations like the National Brain Tumor Society, funding and participating in foundational research that made this breakthrough possible. By securing accelerated approval for a new treatment for H3K27M-mutant diffuse midline glioma (DMG), Jazz Pharmaceuticals has given patients and families the possibility of a new future.

Thank you to everyone at Jazz Pharmaceuticals who contributed to this achievement, and to President and CEO Renee Gala who accepted the Industry Innovation Award from NORD CEO Pamela Gavin and CSOO Kelly Esperias at their U.S. headquarters.

08/21/2026

Did you know Rare Cancer Day is Sept. 23 this year?

Join us Thursday, Sept. 3, from 12-1 p.m. ET for a webinar exploring NORD’s rare cancer policy efforts and ways you can take action with us on Rare Cancer Day 2026. RSVP here: https://bit.ly/4zqpWJX

08/20/2026

Announcing the full agenda for the 2026 NORD Breakthrough Summit: nordsummit.org/agenda

In an unprecedented time for rare disease science, join the patient communities, companies, investors, and officials working to produce new therapies at scale.

08/20/2026

For people living with gastroparesis, everyday activities like eating a meal can come with challenges such as nausea, bloating, vomiting, and feeling full after only a few bites.

During , learn more about symptoms, causes, treatment options, and sources of support in NORD’s Rare Disease Report: https://bit.ly/3VhWXFk

NORD Member IFFGD provides resources and support for people affected by gastroparesis and other digestive disorders, so check them out and give them a follow!

08/20/2026

After years of fighting for answers, including launching NORD member organization the The Jansen's Foundation, Neena Nizar is officially the first patient in an NIH clinical trial for Jansen's disease. This rare disorder is known to affect just 30 people worldwide, three of whom are Neena and her sons.

The study could bring answers and new hope of treatment for her children and others. If it works, the experimental peptide might also help people with kidney failure and hyperparathyroidism.

Read about how Neena made this study happen and join us in celebrating this progress that was decades in the making:

Through sheer determination, Neena Nizar has become the first patient in a clinical trial testing a treatment for her disease — one so rare it affects just 30 people worldwide.

08/19/2026

Today is ! 🌻In recent years, the Malan syndrome community has made great strides, from grassroots fundraising to expanding access to specialized care.

On Rare Disease Day 2025, the Malan Syndrome Foundation provided $50,000 to Children's Hospital of Philadelphia, a NORD Rare Disease Center of Excellence, to help expand its multidisciplinary clinic to increase access to state-of-the-art care for people with Malan syndrome.

And since Rare Disease Day 2024, Jonathan Kowalske, co-founder of Component Brewing Company and father to Mac, who lives with Malan syndrome, has rallied breweries nationwide around Zebra Hop, a special beer benefiting NORD. He has continued to grow the campaign year over year, bringing breweries across the country together to raise awareness and funds for the entire rare disease community.

We’re grateful to the individuals, families, and organizations helping move the Malan syndrome community forward, and we're proud to count the Malan Syndrome Foundation among NORD’s member organizations.

Learn more about this condition, the Malan Syndrome Foundation, and its resources at malansyndrome.org