I just signed a @theactionnet petition: Support the Continuation of the Dog Aging Project. Sign here:

RT @alkeuspharma: FDA Grants Alkeus Pharmaceuticals Breakthrough Therapy Designation for ALK-001 (C20-D3-vitamin A) for the Treatment of St….

RT @newyorkbio: "When we engaged the patient community on their quality of life we learned things that weren't emphasized by physicians or….

#RareDiseaseDay2021

On Rare Disease Day 2021 support the Yellow Brick Road Project.@YellowB20752481

RT @EveryLifeOrg: If you haven’t yet taken the National Burden of Rare Disease Survey – there’s still time -> #burd….

RT @YellowB20752481: Navigating the world can be a challenge for our patients, but they are brave and determined! In "We Move!", see these….

Excited to participate in the 3rd HNRNPH2 Family Meeting 2020 #HNRNPH2 #ybrp #raredisease #Genetics.

I’m excited about the @MassChallenge Virtual Startup Showcase on July 16 & 17 @ 12pm CT/ 11am ET. 200+ #startups from 18 countries & 34 states will present ground-breaking solutions to industry leaders. Join from any internet-connected device. #MC20

RT @GlobalGenes: RARE parents are so powerful. @nytimes features 4 rare families in an article outlining the current landscape of #raredise….

RT @mbeckett: The trillion dollar question. Why are COVID cases increasing while deaths are decreasing? The answer is simple. It's called S….

Congratulations to the MIT100K Finalists and good luck #MIT100K.

Cydan proudly congratulates @Imara for the closing of its IPO. Imara is our second NewCo orphan drug company that was launched in 2016.

Thank you to all who are caring for patients!.

RT @ScottGottliebMD: THREAD: This week tragic reality on ground will overtake events. There are multiple epicenters of U.S. spread. We'll g….

You are in our thoughts !.

RT @ScottGottliebMD: THREAD: On #Coronavirus, we may be entering a period of accelerating cases. The country could look very different over….

Today is Rare Disease Day. As a company focused on accelerating impactful therapies for people with rare genetic diseases, we stand proud with the global community. #RareDiseaseDay

#DYK 72% of rare diseases are genetic and 70% of those rare diseases start at childhood? #RareDiseaseDay

#DYK #RareDiseaseDay