Calling all #pharma / #biotech companies and #PublicHealth philanthropies on the West Coast!

The 2024 #RareImpactAwards are on June 8 in Los Angeles!

Get tickets to celebrate these #RareDisease change-makers on a Universal Studios sound stage with us: rareimpact.org

Welcome new NORD Member Organization, Cure Mucolipidosis, a global organization committed to the identification and treatment of #Mucolipidosis through education, advocacy, and research!

Learn more about this organization and join their patient registry: curemucolipidosis.org

One of our #RareImpactAwards Industry Innovators is Travere Therapeutics, developers of the 1st non-immunosuppressive therapy approved to reduce proteinuria in adults with primary #IgAnephropathy , #IgAN at risk of rapid disease progression.

Meet all the Honorees: rareimpact.org

We're so happy to welcome new NORD Member Organization, @EinstokBorn (Unique Children), the leading support group in #Iceland for kids and young people with #RareDisease s !

Learn how they're overcoming the unique challenges of #RareDisease care in Iceland: einstokborn.is/is/english

We’re thrilled Emmy Award-winning journalist & NBC News Chief White House correspondent Peter Alexander is emcee of our #RareImpactAwards for a second time!

Learn about this year’s stars of #RareDisease innovation and how to attend in LA or tune in live: rareimpact.org

Today we spotlight the 2024 #RareImpactAwards Medical & Scientific Trailblazer, Dr. Jose Abdenur, founder and head of CHOC Metabolic Lab, a NORD #RareDisease Center of Excellence and destination for #metabolic patients from around the U.S.

More: rareimpact.org

Calling all students! Introducing a FREE #RareDisease education course featuring 20 lectures from experts across our 40 NORD Rare Disease Centers of Excellence.

Topics include Careers in Rare Disease, Examples of #RareDisease s, and more. Start learning: bit.ly/4dOUfzo

Join the Organic Acidemia Asn Association and FOD Family Support groups in #Pittsburgh , PA on June 28-29 to connect with other families and learn from medical professionals at the 2024 FOD/OAA Family Forum. RSVP: bit.ly/3Qssq5G

#Acidemia #OrganicAcidemia #OAA #FOD

We're excited to welcome new NORD Member Organization, the 3q29 Foundation, whose mission is to create a path to treatment for chromosome #3q29 syndromes by funding research, increasing access to resources, and building community.

Learn more: 3q29.org

🎳 Calling all #RareDisease individuals and families in the #SouthBend , #Indiana area!

Join Uplifting Athletes & Notre Dame Football for a day of bowling on June 23, 2024. FREE and inclusive for all ages and abilities. Register here: upliftingathletes.org/ua_events/notr…

Connect virtually with other pemphigus and pemphigoid patients and caregivers in the Pacific Northwest area on June 11th. Living with #pemphigus or #pemphigoid is hard enough, but when you feel alone it seems even harder. You are not alone. RSVP: fundraise.pemphigus.org

One of our #RareImpactAwards Industry Innovators is SpringWorks, developers of the first FDA-approved treatment for adults with progressing #DesmoidTumors who require systemic treatment.

Meet all of the 2024 Honorees: rareimpact.org

The Histiocytosis Assoc Association is pleased to announce the 2024 Annual Research Program is accepting grant proposals until June 16 for research into the causes, mechanisms and improved means of treatment for histiocytic disorders. Learn more: histio.org/research/fundi… #histiocytosis

We're thrilled that Abigail Villarreal, this year's Youth Champion Honoree, will perform Ballet Folklórico on stage at the NORD #RareImpactAwards ! Here's an example of her performing the dance.

Tune in live on June 8 or get tickets to join us in LA here: rareimpact.org

One of our #RareImpactAwards winners is Irfan Patel from #Delaware !

A caregiver to kids with methylmalonic #acidemia , and himself recently diagnosed, Irfan got this disorder included in newborn screening and is a Rare Action Network Ambassador and #RDAC member! rareimpact.org

Join the IPPF on June 3rd at 9:00 a.m. PST/12:00 p.m. EST for a webinar on 'Social Determinants of Health and Pemphigus' with Dr. Animesh A. Sinha, Professor of Dermatology at UBuffalo. Register online today: bit.ly/3QSgB9s

#healourskin #pemphigus #pemphigoid

Great news for #Vermont : Governor Phil Scott just signed the Step Therapy reform bill into law - something our Rare Action Network advocates have championed for months!

Vermont joins more than half of states that have taken action to curb #StepTherapy . Learn more: rarediseases.org/vermont-rare-d…

One of our #RareImpactAwards Industry Innovators is Regeneron, developers of the first and only FDA-approved treatment indicated specifically for #CHAPLEdisease , a rare hereditary immune disease.

Meet all the 2024 Honorees: rareimpact.org #chaple

Celebrate the KDA's 25th Anniversary and hear the latest research developments in #KennedysDisease (spinal and bulbar #MuscularAtrophy ) at #KDA2024 , this year's patient-family-researcher conference in Orlando, Florida from Nov. 2-4. Register here: bit.ly/4dLFoWq

From May 26 - June 8, join spinalCSFleak Foundation's #duradash , a community challenge where people participate in any activity appropriate for their current health state to raise awareness about #spinalcsfleak . Check it out here: duradash.causevox.com