Twitter | Search | |
Search Refresh
NORD Aug 2
Want to stay informed on everything NORD is accomplishing? Sign up to receive news and see how we are working to improve the lives of people impacted by . Stay connected:
Reply Retweet Like
Yoni Maisel Jul 29
Reply Retweet Like
PPHF_Asia 2h
Dr. Sanjeeva G N is presenting now on the State perspective & progress on addressing challenges of people living with . He is Associate Prof of Pediatrics, Nodal Officer, Centre of Excellence for Rare Diseases, Indira Gandhi Institute of Child Health, Bangalore.
Reply Retweet Like
PPHF_Asia 3h
Happening Now! PPHF and Takeda are bringing together partners and experts to discuss and examine the needs and priorities of people suffering from especially in the context of COVID-19. Watch us on our Facebook page LIVE:
Reply Retweet Like
Beyond the Diagnosis 16h
August 3rd is CLOVES Syndrome Awareness Day! Meet Victor. Victor loves to run, jump, and climb. He loves pizza, cookies, and cake! Victor's family call him their little Superhero!
Reply Retweet Like
Child & Youth Care 20h
It's WOMEN'S MONTH! August we are focusing on women, starting off this week with Female Hemophilia. If you have a mother/sister/daughter/ aunt/ niece/grandmother who might benefit from these articles, please share.
Reply Retweet Like
NORD Jul 29
Interested in learning more about 's signature charity running program-? Runners are paired with rare community members to raise awareness for while fundraising to support patients. More:
Reply Retweet Like
Rocket Pharmaceuticals 19h
that is a technique that inserts the functional copy of a gene into a patient’s cells to treat or prevent genetic diseases? Learn more about our pipeline & the safe & potentially curative we’re developing for :
Reply Retweet Like
FulcrumTx Jul 30
Today is ! We can't wait to be back together in the office as a team as we work to improve the lives of those living with .
Reply Retweet Like
Chiasma Pharma 17h
We never give up. Resiliency is in our DNA. Learn more about our focus to improve the lives of facing challenges associated with injectable therapies for their :
Reply Retweet Like
Neutropenia Network Aug 2
Dash is a beautiful boy. Unfortunately he is suffering from Autoimmune Neutropenia. We are here with the story of Dash. See here his journey - Thanks Cure The Count!
Reply Retweet Like
Raquel Peck 🇧🇷 🇬🇧 Jul 30
Brilliant meeting today. It’s so great & inspiring to hear about the amazing work the orgs are doing to advance the science in their disease areas. More to come tomorrow!
Reply Retweet Like
Scleroderma Foundation Colorado Jul 30
Rare disease drove two women on a mission to change FDA drug review process
Reply Retweet Like
SunoIndia 4h
An estimated 100mn Indians are said to suffer from any of the over 6000 . So how is it that the conversation is so limited and restricted to only those who get impacted? Tune into by to know & stay tuned for Season2! 🎶
Reply Retweet Like
Solve-RD Jul 31
Check out the poster & infographic illustrating the diagnostic odyssey many people affected by experience. Created through the -led Community Engagement Taskforce within the project presented at & 👉
Reply Retweet Like
NYAS 18h
Monday, August 10th is the deadline to submit abstracts for the Award Symposium later this fall. Please submit your abstracts here:
Reply Retweet Like
Sean Ekins Jul 31
our new paper with UNC catalyst group on chordoma is out and open
Reply Retweet Like
Foundation to Fight H-abc 17h
Hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC) is a rare genetic disorder that affects certain parts of the brain, specifically the white matter, which affects one's motor abilities. Learn more:
Reply Retweet Like
Dr. Iván Martínez Duncker MD ScD - PI - 19h
These cells (fibroblasts) obtained from a skin biopsy of a potential patient give us hope in identifying the affected gene and ⬆️ knowledge about these . Our goal is to give long-awaited answers and a chance to improve his quality of life.
Reply Retweet Like
Jale Şahin Jul 28
Patients of Rare Diseases and their families have a long road for the diagnosis of their ilnesses. In each step of their journey, they need solidarity, support and faith into the healthcare system.
Reply Retweet Like