Board member | Non-profit Leader | Consultant | Rare Diseases | Patient Advocacy I Former Executive Director NTSAD.
Boston, MA
Joined December 2013
- Tweets792
- Following425
- Followers450
- Likes981
Sue Kahn retweeted
This #IWD, we are celebrating the achievements of women pioneers at LogicBio & in the broader #biotech space who have paved the way for future generations of women and girls to lend their vision and help build a healthier, more equitable society. bit.ly/3vyTSoW
Sue Kahn retweeted
USA today feeatured an amazing NTSAD family. Read about Jessie here.
msn.com/en-us/news/us/rare-d…
Support rare families this #GivingTuesday. bit.ly/3m3jz9k
Sue Kahn retweeted
We’ve received FDA Fast Track Designation for our industry-leading #GeneTherapy, AXO-AAV-GM1, for the treatment of #GM1 gangliosidosis. We’re working diligently to bring hope to children & families affected by this rare, terminal disease. Read more: bit.ly/3B2xZgE $SIOX
Sue Kahn retweeted
#GM1 Type 1 is the most severe form of the disease. Children with Type 1 typically present symptoms around 6 months of age when development may begin to slow & muscles may become stiff/weak with an eventual loss of motor skills. Learn more: bit.ly/3ggI7uV @ASGCTherapy
Sue Kahn retweeted
"There needs to be a sense or urgency within the community and for researchers for treatment to be found." Cure Darian is streaming on the #GGSummit now with The Disorder Channel. #careaboutrare #raredisease @curegm1foundation #GM1Gangliosidosis #curegmi #GM1
Sue Kahn retweeted
It took 10 months for Embree's parents to get a diagnosis of #Sandhoff disease for their daughter. To read how an early diagnosis could have made a difference for their family and other rare families' journeys to diagnosis profiles, visit bit.ly/2YHYnPD
#MaketheRareDx
Sue Kahn retweeted
Today, we announced the dosing of the first Type I (early infantile) patient, the most severe form of #GM1 gangliosidosis, in our ongoing study of AXO-AAV-GM1 #GeneTherapy. We are continuing to screen and enroll Type I patients. More here: bit.ly/3E1taH7 $SIOX
Sue Kahn retweeted
Thank you to our partner @TayshaGTx for raising awareness of #TaySachsDisease!
September is #TaySachsAwareness month. Tay-Sachs disease is a rare, genetic neurodegenerative disorder of the central nervous system (CNS) that can impact children within the first few months of life and for which there are no approved therapies. Learn more.
Sue Kahn retweeted
With Sue Kahn departing after 14 incredible years, NTSAD is searching for its next Executive Director. We are seeking a passionate, experienced, and inspiring leader in this unprecedented time of progress and hope. Learn more and apply! #NTSAD bit.ly/3dWqPBW
Sue Kahn retweeted
What a great start to the conference. Thank you Sean and Kyle @2DDPodcast for kicking off the conference with your warm and thoughtful conversation and thank you to our panelists and everyone for tuning in! #NTSADafc2021 #hearttohearthometohome!
Sue Kahn retweeted
Help turn the internet blue on 4/18/21 exactly one year since Maxie left this earth. Bright blue was his favorite color & we're inviting everyone to wear blue & tag us & share pictures on social media: hashtags:
#MaxieForever
#RememberingMaxie
#CanavanAwareness
#CureCanavan
Sue Kahn retweeted
Kevin Levine has been raising awareness about the importance of carrier screening ever since his daughter Lila died of Tay-Sachs disease in 2001. Read more about Kevin’s story now in his recently published article for the Columbus Dispatch. bit.ly/3m79nOd
Sue Kahn retweeted
.@NTSAD's Annual Family Virtual Conference is April 22-25. The conference is free and open to any family coping with a #TaySachs, #Canavan, #GM1 #Gangliosidosis, or #Sandhoff disease diagnosis, members of industry, researchers, clinicians & rare allies. bit.ly/2McA6eI
Sue Kahn retweeted
#TaySachs & #Sandhoff diseases are pediatric neurodegenerative lysosomal storage disorders resulting from mutations in beta-hexosaminidase, a key enzyme in the lysosome. Read more about our developing pipeline here: bit.ly/2PhsA3l
Sue Kahn retweeted
Watch Sheryl Sandberg, founder @LeanInOrg, Facebook COO, co-author of Option B: Facing Adversity, Building Resilience, and Finding Joy discuss the challenges and emotions experienced when facing life-altering, unexpected situations w/ the NTSAD Community. bit.ly/2PI1VN8
Sue Kahn retweeted
At Sio, we are all patient advocates. At our latest company town hall in honor of yesterday’s @RareDiseaseDay, we had the privilege of hearing from a family impacted by #TaySachs. We were proud to support @NTSAD during the event by wearing their Rare Bear shirts! #GM1 #Sandhoff
Sue Kahn retweeted
As @NIH celebrates @rarediseaseday, check out today’s session at 11:50 ET with Dr. Cynthia Tifft, Deputy Clinical Director @genome_gov, #GM1 expert & our clinical trial investigator, as she discusses hope in #GeneTherapy for #RareDiseases. More info: ncats.nih.gov/news/events/rd…
Sue Kahn retweeted
NTSAD acknowledges the entire Rare Community for its tireless efforts to advocate, raise awareness, and fund research. Together we work to find effective treatments and to support and honor rare, loved ones. #RareDiseaseDay
Sue Kahn retweeted
Did you know that since 1957, NTSAD has provided support, community, and connection for Rare Families, and today specifically for families living with Canavan, GM1, Sandhoff and Tay-Sachs? #NTSADCaresForRare #RareDiseaseDay