Our 2024 #RareImpactAwards Youth Champion—the first ever—is Abigail Villarreal, #phenylketonuria ( #PKU ) advocate and student from #SanAntonio !
Abby created “Be Rare Spirit Day,” a high school event that will continue on after she graduates. Learn more: rareimpact.org
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
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
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
With #MothersDay tomorrow, we're spotlighting one of this year's #RareImpactAwards Community Champions, grandmother and rare advocate Darlene Shelton of Danny's Dose!
Darlene is transforming emergency care for #RareDisease patients with patient-carried medications... 🧵
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
One of our #RareImpactAwards Industry Innovators this year is Pharming Group NV, developers of the first approved treatment for Activated PI3K Delta Syndrome ( #APDS ), a rare primary immunodeficiency.
Meet the rest of our 2024 Honorees: rareimpact.org
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 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
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
Today on #NationalDNADay , we are spotlighting Jana Monaco, one of this year's NORD #RareImpactAwards Honorees whose family's story is profoundly impacted by DNA. 🧵
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
Thank you to NORD for recognizing SpringWorks for developing the first approved therapy for adults with #DesmoidTumors . We look forward to celebrating the advances being made on behalf of patients with #RareDiseases at the #RareImpactAwards in L.A. this June.
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
Announcing our 2024 NORD #RareImpactAwards Industry Innovators!
These companies have developed groundbreaking FDA-approved #RareDisease therapies that brought hope to many in our community.
Meet the Industry Innovators of 2024: rareimpact.org
We're thrilled to announce the 2024 NORD #RareImpactAwards Honorees!
These stars have each made incredible advancements on behalf of the #RareDisease community, and we look forward to celebrating them this June in Los Angeles!
Meet the Honorees: rareimpact.org
Today we spotlight Policy Changemaker + #RareImpactAwards Honoree, #Minnesota Rep. Liz Reyer! @lreyer has been a staunch ally to constituents with #RareDiseases , including establishing and serving on the MN #RDAC .
Meet all our #RareImpactAwards Honorees: rareimpact.org
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
The #RareImpactAwards celebrate individuals, organizations and innovators who have made strides to benefit the #RareDisease community.
Nominations for the 2024 Rare Impact Awards are NOW OPEN! Nominate your rare hero today: bit.ly/3HvpEYM
The 2024 #RareImpactAwards will honor the companies pioneering advancements in #RareDisease treatment.
One of those is BioMarin, developers of the 1st FDA-approved gene therapy for adults with severe #hemophiliaA .
Meet all of the 2024 Honorees: rareimpact.org
