Happy #MothersDay to all the #RareDisease moms and grandmas out there! Thank you for all you do to advocate and care for your kids.

To those living with their own #RareDisease s, we honor you today and support you all year long. Enjoy the day with (or without) your families! 💐

One of our #RareImpactAwards Industry Innovators is Ipsen, developers of the 1st treatment approved to reduce extra-skeletal bone formation in people with #FibrodysplasiaOssificansProgressiva ( #FOP ).

Meet all the 2024 Honorees: rareimpact.org

NEW: NORD launched the first internationally-based patient registries on our #IAMRARE platform!

Based in #Canada for Canadian #PKU and #MPS patients, they represent a unique collaboration with the CHEO Ottawa Research Institute, CanPKU+, and CanMPSSociety. rarediseases.org/first-canadian…

In 2023, #Georgia and #NorthCarolina expanded Medicaid access, enabling more people with #RareDiseases to get care. However, 10 states STILL have not expanded #Medicaid eligibility. Is your state one of them?

Find out in the NORD State Report Card, here: bit.ly/3RDI1yF

Meet one of the Policy Changemakers of this year's #RareImpactAwards , Florida Gus Bilirakis.

As Co-Chairman of the #RareDisease Caucus, Rep. Bilirakis advances the understanding that rare diseases are not a rare problem but a shared problem. Read more: rareimpact.org

Yesterday, NORD hosted a workshop on #RareDisease Clinical Trials at the ASGCT Annual Meeting!

Thank you to everyone who joined us to learn about how to design effective clinical trials and the importance of the patient voice throughout drug development. #ASGCT2024

FDA approved a 2nd gene therapy to treat #HemophiliaB ! Gene therapies offer hope for the more than 80% of rare diseases that are genetic.

Congrats to Pfizer Inc. & NORD patient advocacy members HemoSoCal, National Bleeding Disorders Foundation & Hemophilia Federation of America (HFA) on this accomplishment. #MoreTreatments4Rare

📢 New Research Grant Announcement!

The Patient-Centered Outcomes Research Institute's new Addressing Rare Diseases PFA offers up to $100M in available funding for patient-centered #CER projects on symptoms management, timely diagnosis, or improving care delivery for #RareDiseases .

Learn more: pcori.me/3SFCmJl

Poster abstract submissions for the 2024 #NORDSummit are open until June 12!

Submit your research for the chance to be featured at this high-profile #RareDisease and #biotech event in October: bit.ly/3LiYAw8

The FD Foundation FD Foundation is happy to announce an #RFA for an Early Stage Career Investigator #grant funded by the Foundation’s Clare & Philip Wexler Research Fund. Letters of Intent are due by June 14. For info, visit: familialdysautonomia.org/esci

#FamilialDysautonomia #Dysautonomia

Join NORD and Medlive for 8 easy-to-digest, on-demand #CME modules covering gene and cell therapies for several relevant disease states. Expert clinicians lead each module and review relevant data.

Start here: bit.ly/3w56Qxu

#CellTherapy #GeneTherapy

We're proud to spotlight the winner of the Abbey S. Meyers Leadership Award at this year's #RareImpactAwards , HemoSoCal! This 70-year-old organization fosters critical engagement among Spanish-speaking communities affected by bleeding disorders.

More: rareimpact.org

Today NORD celebrates 41 years! 🎉🦓

We look forward to continuing to improve the lives of everyone with #RareDiseases and those who love us by driving advances in care, research, and policy - under the leadership of new CEO, Pamela Gavin! Read more: bit.ly/4aaNCEh

One of our 2024 #RareImpactAwards Industry Innovators is Chiesi Global Rare Diseases, developers of the first FDA-approved enzyme replacement therapy for non-central nervous system manifestations of #alphamannosidosis .

Meet the rest of this year's Honorees: rareimpact.org

We're thrilled to share the winner of our Living Rare Design Contest, Jessica B.! Her illustration shows that having a #RareDisease makes someone special.

To get a tote bag with this design, join us at the NORD #LivingRareForum in Los Angeles, June 7-8: livingrare.org

We joined NORD Member, PALS SkyHope, at their event in Boston about 'Connecting Flights to Care.'

Long-time friend of NORD, PALS SkyHope provides free air transportation to attend medical appointments more than a two hour drive from home. Learn more at palservices.org