The Rare Moments
You know those rare moments that give you goosebumps? The ones that provide a new perspective, connect two ideas in a way you’ve never considered before, or just really hit home?
For Rare Disease Day this February, you’ll find a curation of those moments here — The Rare Moments. Patients, families, researchers, and people working to advance rare disease treatments will be using this space to pass on their goosebump moments. From past moments, to the significant highlights of the 2024 Rare Disease Week and Rare Disease Day, and even hopes for the future, here you will find diverse and valuable insights that matter to our community and expand our reader’s understanding of what it means to live with and work with rare diseases.
Be sure to check back regularly throughout the month to catch the latest posts and submit your Rare Moment for consideration!
See what’s been shared
That’s a wrap for now!
This feed is no longer active or accepting submissions, but we encourage you to check out the posts below which were submitted during February 2024 in support of Rare Disease Day.
Resources
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!
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.org/about-us/our-events/tnbc-day/virtual-events-2024TNBC Day | Triple Negative Breast Cancer Foundation
Every day in March is TNBC Day! Triple Negative Breast Cancer Day is the only global event dedicated to raising both awareness and funds exclusively for triple negative breast cancer. Find out how you can get involved!
tnbcfoundation.org
www.linkedin.com/posts/canaryadvisorsllc_raredc2024-rarediseaseday-activity-7163158752681000960-1zXJ?utm_source=share&utm_medium=member_desktopCanary Advisors® on LinkedIn: #raredc2024 #rarediseaseday
Next up, we'd like to introduce you to Carolina Consuegra, a wonderful advocate and talented medical writer. Science2People #RareDC2024Â #RareDiseaseDay.
www.linkedin.comRead our latest blog post to learn about the cell and gene therapy trends Trinity Life Sciences is watching for 2024!
url.uk.m.mimecastprotect.com/s/qBzUCRojNiVXNLnt98iUV?domain=bit.lyThis Rare Disease Day, I am thinking about how phenomenal it is to see how rare oncology patients have benefitted from the 6 CAR T treatments now available! It is exciting to watch how continued advancements have turned CAR T into a new development paradigm for autoimmune diseases – looking forward to seeing these additional patient communities benefit from cell therapy!
In celebration of Rare Disease Day, I am excited to share our most recent draft class from the 2024 Young Investigator Draft! This year we highlighted the significant potential of research in this space when powered by the platform of sports—especially as we surpassed our $1 million in total funding granted. You can watch a recap of the event at the below link!
When my daughter was diagnosed with WAGR syndrome, none of the doctors that were treating my daughter knew anything about WAGR. Since that time, I’ve dedicated my time and energy to advocate for my daughter, spreading awareness of WAGR throughout my local and the international communities.
Wagr.orgInternational WAGR Syndrome Association
The International WAGR Syndrome Association is a not-for-profit organization comprised of an international network of families, health care professionals and researchers, and others who care for individuals with WAGR syndrome.
wagr.orgRare diseases affect hundreds of millions of people globally, presenting a massive challenge to health equity. Our team is focused on changing that sad reality for patients across many therapeutic areas. From rare cancers such as the one that took my sweet cousin Ryan last year to rare cardiovascular diseases, I’m proud of how hard our teams work every single day to improve the standard of care for rare disease patients. Their passionate and purposeful commitment to saving and improving lives is not just a job; it’s creating meaningful change for our loved ones, their caregivers, and for humanity as a whole.
With extensive commercialization expertise and deep knowledge of rare diseases, Trinity Life Sciences has supported the launches of some of the most impactful drugs and therapies globally. We remain committed to helping our clients overcome unique commercialization challenges faced by specialized treatments and strive to create a better future for those living with rare diseases.
Jim Geraghty said it best today at the 9th Annual Rare Disease Day Event at the Broad Institute. “It’s a marathon, not a sprint,” when it comes to rare disease research. Always a science-focused and inspiring event at the Broad!