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Children and adults with X-linked hypophosphatemia #XLH wanted for research study of a new treatment regimen! We’re studying the effect of a drug called calcitriol on bone health, without phosphate supplementation. Participants will be reimbursed for travel expenses.

Today, the Senate passed a Continuing Resolution that includes a 57% cut to the Congressionally Directed Medical Research Program. While the CR may keep the government running in the short term, it brings with it concerning implications for #Duchenne and #Becker research.

Want to know a secret? The 10,000 rare diseases are the world’s largest dataset of “unpublished biology” and the biggest opportunity space in medicine. (And guess what? Rare diseases are not on China’s radar…)

The world lost a beautiful and bright angel today. No words. Heartbroken. Please pray for our dear friend Tracy Dixon-Salazar n her family as they process this unimaginable loss.

The HHS.gov eliminated thousands of positions across federal agencies including the FDA, CDC, the ACLgov, and NIH. While full details of the roles and divisions impacted are still being assessed, we know that our landscape has significantly shifted today. 𝗢𝘂𝗿 𝗿𝗮𝗿𝗲

Reauthorization of the Priority Review Voucher will unlock the $1T+ rare diseases opportunity

The real miracle isn’t drug discovery — it’s that rare disease families keep showing up to push science forward, even when the system barely notices they exist. #MakeAmericaCareAboutRare Secretary Kennedy

Thank you Effie Parks for so beautifully capturing our journey to chane the narrative for Jansen's disease.

"Medicaid isn’t just a policy, it’s what makes hope possible." For Sky Collins and her family, #Medicaid is a lifeline that helps their daughter live with independence, dignity, and connection through #HCBS Waivers. But proposed Medicaid funding cuts could threaten this vital

We at the FDA now have to ask big questions that we’ve never asked before. Why does it take over 10 years for a new drug to come to market? Why are we not modernized with AI and other things? We’ve just completed our first AI-assisted scientific review for a product and that’s

.Dr. Martin Makary Vinay Prasad MD MPH, as a physician living with ALS, I urge you to grant NurOwn accelerated approval to break a 156-year cycle of despair for a 100% fatal disease. ALS patients, with a median survival of 2–3 years, cannot wait 5+ years for Phase 3b trial results,

The Energy and Commerce Committee Committee continues budget reconciliation work, including potential Medicaid changes. Watch the livestream, patient stories are being shared to give a face to Medicaid participants. youtube.com/watch?v=_FHQYx… Your story matters! Share your Medicaid experience &

You’re in great hands America! Here are the 2025 Harvard/MIT MD-PhD graduates who have dedicated their lives to providing the best care to your families & delivering you medical cures from their groundbreaking science. NIH Jay Bhattacharya, MD, PhD HHS.gov Secretary Kennedy Harvard Medical School Massachusetts Institute of Technology (MIT)

The Weight He Carried: How One Rare Dad Turned Heartbreak into Horsepower w/ Jeff Allen I cried when I first interviewed Jeff. To be honest, I expected a polished media chat - maybe even a PR rep listening in, which usually takes some of the magic out of it. He’d just won $10

We find that PTH1R/Gas/PKA/SIK3/HDAC4 activation drives the growth plate phenotype and show that an inverse agonist can correct lab abnormalities Wein Lab Center for Musculoskeletal Research MGH Endocrine Unit Research Mass General Brigham Research Pedro Brugarolas Gellman Lab Neena Nizar

If you’re a scientist/doctor and your grant/startup funding has suddenly dried up, then apply for a job at U.S. FDA and shape the future of medicine and especially rare diseases

This gives us hope. Rare diseases need treatments now. We need new pathways and accelerated routes to trials. #RareDiseaseTruth

Father’s Day may feel especially heavy this year. Whether you're missing someone, grieving, or simply trying to hold it together - we see you. You're not alone. Sending love and strength to everyone navigating today. 💙 #FathersDay    #WeSeeYou #KeepFamiliesTogether #EndWar

An NIH staffer reacts to today's ruling: "I'm looking forward to the day that we are so slammed with work trying to reinstate everything that we had to terminate illegally — I'll work 24/7 to make that happen if I can." My story for nature nature.com/articles/d4158…

We need to think and talk about rare genetic diseases the way we think and talk about rare earth metals: Precious, hard-to-find, incalculably valuable. The raw materials that will fuel epic technological advances for generations to come.