Speakers in #RDDNIH session 1 highlighted the importance of finding and connecting existing data to help the #RareDiseases community understand rare diseases and advance research. #RareDiseaseDay

“It’s time to move away from only natural history clinical trials as we work toward first-in-human studies.” - James K. Gilman, MD #RareDiseaseDay #RDDNIH #RareDiseaseDay2023

“Diversity in #genomics is not just good for the African population. It’s good for all of us… It’s even more important to think about the growing diversity of our country.” — Tshaka J. Cunningham, Ph.D. #RDDNIH #RareDiseaseDay

Happening now at #RDDNIH ➡️ Session 4: #RareDiseases Therapeutics and the Role of Advocacy and Industry Collaborations ✅ Featuring Kristen Wheeden, MBA, Rare Disease Caregiver; CPAG Representative, #Porphyrias Consortium (PC) Join the livestream: videocast.nih.gov/livew.asp?live…

Truly phenomenal news and so special to hear this at the end of #RDDNIH by Ron Bartek. There were so many happy tears in the room. #HopeIntoAction

Wow -this is a very impressive showing of #RareDisease #advocacy superheroes!

“It’s not rocket science, but to do the science we need the rockets.” – NCATS Director Joni Rutter The tiny technology held by Dr. Rutter is an example of the #TissueChips that will head to the International Space Station to speed research on health and the development of new treatments.

NLM was proud to light up for #RareDiseaseDay & even prouder to support NCATS's vision of more treatments for all people more quickly. In this week's blog, JoniRutter discusses how #DataScience tools will speed #RareDisease disease solutions. nlmdirector.nlm.nih.gov/2023/03/15/dat…

"It's not rocket science, but we do need rockets to do the science," JoniRutter on NCATS collaboration with NASA to discover new insights into the aging process.

Catch up with CRISPR TODAY at 12pm (ET)❗️ #SCGE PI ErikSontheimerLab joins CRISPR pioneers for a free webinar highlighting the current opportunities and challenges for #CRISPR technology. Register ⬇️

Collaboration is at the heart of what we do at NCATS! Hear from NCATS’ Matt Hall, Ph.D., about what could be next for #DrugRepurposing or #DrugDevelopment in collaboration with the Bardeesy Lab at MassGeneral News. 👀

#DYK that the #SBIR & #STTR programs are one of the largest funding sources for non-dilutive capital for early-stage #lifesciences #smallbiz? Learn more about these programs today: ncats.nih.gov/smallbusiness #NCATSsbir

Join our team! We're hiring a director for NCATS’ Office of Strategic Alliances. The candidate will manage the technology transfer program & serve as an advisor to NCATS leadership on matters related to NCATS’ #SBIR and #STTR programs, patenting & more. bit.ly/3leKO5I

#CTSAProgram researchers redrew United States maps of three #FungalInfections and found they have spread outside their usual regions of infection, causing severe and life-threatening infections. Learn more about the study findings: bit.ly/3xILxPq

#Clinicaltrials research the way a drug or treatment will interact with the human body, and are critical for determining safety and efficacy. Learn about what it's like to participate in a #genetherapy clinical trial ⬇️

Friendly reminder that if you have thoughts about the current state of postdoctoral research training and career progression within the biomedical research enterprise, the NIH would like to hear from you by April 14, 2023 📝💻bit.ly/3lDU2s0

P.J. Brooks, Ph.D., Acting Director of the Office of Rare Diseases Research at the National Center for Advancing Translational Sciences (NCATS)NCATS, describes the two main types of gene therapy currently in development. Rare Diseases Clinical Research Network checkrare.com/ex-vivo-vs-in-…

Just so we are clear…it wasn’t me, but our EO has #setthebar! NCATS

Somatic Cell Genome Editing (SCGE) Outreach Fall meeting in Bethesda. Great opening by Walter J. Koroshetz highlighting some progress over the last year NIH Common Fund #SCGE program with several #FDA meetings to advance #genetherapies. Right now we are hearing advances for #prenatal #geneediting, exciting data.

The #AssayGuidanceManual Webinar featuring Nobel laureate Dr. Carolyn Bertozzi is tomorrow! Don’t forget to register and tune in at 11 a.m. EDT to hear more on New Therapeutic Modalities Inspired by Glycoscience: sites.google.com/ncats.nih.gov/… #Glycoscience