The Rare Moments
Every color tells a story, and we want to hear yours — the stories that offer fresh perspectives, inspire hope in the community, connect unexpected ideas, or simply resonate with you on a personal level.
For Rare Disease Day this February, you’ll find a curation of those stories here — The Rare Moments. Patients, families, researchers, and those dedicated to advancing rare disease treatments will be using this space to share their rare stories. From past experiences with lysosomal diseases to significant highlights of the 2025 Rare Disease Day, and even hopes for the future, you will find diverse and valuable insights that matter to our community and expand our understanding of what it means to live with and work with rare diseases.
Be sure to check back regularly throughout the month for the latest posts, and don’t forget to submit your colorful Rare Moments!
See what’s been shared
Resources
Amplifying Patient Voices to Understand & Better Serve the MPS Community
Infographic
Incorporating the Patient’s Voice
White Paper
How To Build Mutually Beneficial Relationships with Advocacy Groups
Insights
How Worldwide and Every Cure Are Using AI To Unlock New Rare Disease Treatments
Insights
Meet Your Rare Disease Team
Fact Sheet
Rare Disease Day
rarediseaseday.org
Glut1 Deficiency Foundation | Research Compass
g1dfoundation.org
Workshop: Qualifying Biomarkers to Support Rare Disease Regulatory Pathways
reaganudall.org
Galactosialidosis Network
galactosialidosis.org
Every Cure
everycure.org
Uplifting Athletes
upliftingathletes.org
We live for the rare moments.
Thank you for sharing and reading this curation from our rare disease community!
One of the families in our community had an event annually for 10 years that was a 3 day hangout session on a farm for FA families. People camped out, and there were all kinds of activities, such as swimming, fishing, yoga, tie-dying, crafts, fireworks, and all-you-can-eat ice cream. At this event, people with Friedreich’s ataxia (FA) never felt out of place, and we were all able to be together without the awkwardness that can be present around people who might not understand the experience of FA.
My Rare Moment is anytime I get to be with my people.
curefa.orgFARA – Friedreich's Ataxia Research Alliance
The Friedreich’s Ataxia Research Alliance (FARA) funds and facilitates research to slow, stop, reverse, and cure Friedreich’s ataxia. FARA is a key resource for both FA families and researchers.
curefa.orgI have seen so many families without hope losing children (young and adults) to rare diseases despite the investment of time/money, and emotions. One of the most fulfilling days for me was when one such family was able to watch their child markedly improve about 8 months after treatment!
This year, I had the opportunity to facilitate roundtable discussions at both World Orphan Drug Congress (WODC) Europe and WODC United States. These discussions explored operational challenges and ethical barriers surrounding genetic testing, and I was fascinated to discover what varied conversations the different regions had. While WODC Europe centered heavily on ethical considerations around genomic and genetic data, WODC U.S. was highly operationally focused.
With Rare Disease Day in two weeks, I feel so fortunate to help further these conversations around genetic testing, which can make a significant impact in the treatment of rare disease patients around the world.
Check out my blog to learn more about the key regional distinctions:
www.worldwide.comWorld Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing
Explore insights from the World Orphan Drug Congresses in Europe and the U.S., where Worldwide Clinical Trials’ Derek Ansel led discussions on operational challenges and ethical barriers in genetic testing for rare diseases.
www.worldwide.comWith Rare Disease Day coming up, I’m reflecting on the continued innovations in CAR T treatments for rare oncology patients over the last few years. The global research community launched a record number of CAR T clinical trials in 2024, gaining momentum while building on the strength of several approved CAR T treatments. I’m excited to see these treatments now developed for rare patient communities outside of oncology and looking forward to being part of setting these new standards of care.
www.worldwide.comFind out more about bringing CAR T to new clinics in my on-demand webinar:
Expanding CAR T Beyond Oncology: Medical, Operational & Practical Considerations
Watch this on-demand webinar on expanding CAR T therapies beyond oncology, with insights for autoimmune and neurology trials for sponsors, sites, and patients.
www.worldwide.comHear from Nathan Chadwick, Senior Director and Therapeutic Strategy Lead for Rare Disease.
youtu.beWe are so excited to re-launch Rare Moments, our website dedicated to sharing the stories and experiences of the rare disease communities throughout the month of February in honor of Rare Disease Day. Please use this safe space to share your memories, notes, events, thoughts, and resources so that we can help elevate each other’s voices and connect like-minded individuals. So much happens during and ahead of Rare Disease Week, so bookmark this page and visit often to stay current on everything rare disease!
We’re excited to relaunch Rare Moments—a space to share updates, events, and resources throughout the month of February. Join us in amplifying voices and stay connected by bookmarking the page!
I had such a fulfilling experience visiting my members of Congress as part of Rare Disease Week on Capitol Hill. My fellow advocates and I raised our voices to encourage their support of policies that benefit rare disease patients. Hearing everyone’s stories was so inspiring.
We are proud to support and bring awareness to Triple Negative Breast Cancer Day which was yesterday, March 3rd. Individuals in the United States have a 1 in 8 chance of developing breast cancer and TNBC accounts for 10-25% of those breast cancers. Me and many others at Worldwide are excited to take part in the many events occurring throughout March that bring awareness to this disease. Please join us at any of the highlighted events listed below.
tnbcfoundation.orgCheck out the Minnesota Twins’ Target Field lit up for Rare Disease Day!