Twitter Beğeni Hilesi

We hosted a meetup of #StudentsForRare leaders from schools and universities around the country here in DC before tomorrow's #NORDSummit. The future of #RareDisease advocacy depends on the folks in this room! Join Students for Rare: bit.ly/3LNJjqh

95% of #RareDiseases patients have no FDA-approved treatment. Any law or policy that discourages companies from doing the research necessary to add an indication for a #raredisease (such as the Inflation Reduction Act) supports the unacceptable status quo. #NORDSummit

Dr. Robert M. Califf Califf an article by science writer Ed Yong in The Atlantic: “Technological solutions also tend to rise into society’s penthouses, while epidemics seep into its cracks.” #NORDSummit 3/

"Researchers often think they know the most meaningful endpoint, but that can be different for patients as a whole, but also for individuals within your community. Understanding the patient perspective is the first step." - Heidi Bjornson-Pennell, Chan Zuckerberg Initiative #NORDSummit

.heidi bjornson of Chan Zuckerberg Initiative: There’s too much to be done to duplicate effort. Do a landscape & identify the gaps you can fill. Be a central provider of resources & convene researchers with patients to align shared priorities to drive #raredisease research. #NORDSummit

As I listen to panel, I wish we had time to discuss a difficult concept: There is not "a" patient voice. There are patient voices. They don't always agree. I hope that FDA, sponsors don't insist on consensus before they will work w them. Diverse opinions can help. #NORDSummit

Fact: ALS caregivers typically don't take care of their own health. They don't do annual checkups, they don't have their own concerns addressed they don't go to the dentist, ... You get the picture. 1/

Got hockey? It’s gonna be a GREAT Saturday. Check out this action-packed event all benefitting rare disease. This Saturday the fun lasts all day. KIF1A.ORG, Global Genes, #RareAsOne. If any org wants to spread awareness and have a table at the event, come on out! All communities

Grateful for the rare disease warriors in the space who continue to be the voice of change for so many. ♥️

What is a myth or misconception about living with a #RareDisease or #ChronicIllness that you still see spread? Let's combat the myths right here.

Two years ago @CBSSundayMorning came to our house to talk about the ALS movement. This summer, they came back to talk about our success, our new projects and our lives. Tomorrow morning Lee Cowan’s piece will air as part of CBS Sunday Morning. I truly hope you will watch it!

Registration is open for our #FDARareDiseaseDay event on March 1. Join us to hear from experts on empowering patients and health care professionals with key information and working to advance medical products for rare diseases. fda.gov/news-events/fd…

💰GIVEAWAY DAY 8💰 Is there any better gift than CASH?? For a final giveaway we’re adding $250 to one lucky winner’s Cash Back balance 🤑 To enter: -Follow Rakuten on X -Retweet this post -Tag up to 5 friends in the replies (1 reply = 1 entry)

Love the willingness to listen to & learn from #raredisease community—> “More often than not, patient groups are teaching us,” says Jodi Rutter of NCATS at EveryLife Foundation Community Congress

📣 To WIN a FREE OCEANFRONT 2 Room Suite May 2nd-May 6th, 4 nights: 1. FOLLOW us on X. 2. Re-Tweet as many Adventurer Tweets as you want 😉. 3. LIKE this Tweet. Wildwood 365 Wildwoodboardwalk Watch the Tramcar Please #wildwoodnj #nj #wildwoodnewjersey #wildwood365 Rules next Tweet

#SCN2A Fact of the Day Knowledge is power, & sharing these insights, we can create a more informed & supportive community. Read, learn, & share because awareness is the first step towards understanding & change #SCN2ADay24 #SCN2AAwareness #GlobalAdvocacy #cureSCN2A #HopeForACure

Enormously proud and honored to do this important work. ❤️

.NORD to host a webinar for parents & other caregivers of teens with #RareDiseases on February 29 at 7-8pm ET which will include mindfulness exercises plus discussions of grief, radical acceptance, boundaries & self-advocacy rarediseases.zoom.us/meeting/regist…. #RareDiseaseDay