Top Tweets for #Sandhoff
A clinical study of gene therapy for GM2 gangliosidosis shows encouraging results at @UMassChan: https://t.co/Ypa8ZlUwI1
“Biochemically it worked.” - Heather Gray-Edwards, DVM, PhD
#GeneTherapy #TaySachs #Sandhoff @NatureMedicine @UMassMemorial @NTSAD @BluGenesFdn @UMass
"Offering an FDA-approved drug for off-label use could provide hope and improve both life expectancy and quality of life for these patients."
#Sandhoff #TaySachs #lysosomalstoragedisorders
https://t.co/hCf1bJfJfw
Congratulations @KTsoNeuro! Functional recovery and rescue of many disease phenotypes in #Sandhoff mice using combined bone marrow transplant and CSF1R inhibitor-induced repopulation to replace #myeloid Hexb-deficient cells with Hexb-sufficent cells
Excited to share the first preprint from my PhD work! We hope that this work will be illuminating both for the treatment of lysosomal storage disorders and for our understanding of microglial biology in homeostasis.
https://t.co/VFTXgiaXgE
We're proud that Patricia will be presenting 2 posters at #ECRD2024 on behalf of the charity this week! The poster was developed with @Cognitant and is about our Clinical Toolkit for GM2 -you can read it in full here 👉 https://t.co/Lq93qB6D6w #catsfoundation #taysachs #sandhoff
On our next Charity Partner spotlight - CATS Foundation.
The Cure & Action for Tay-Sachs (CATS) Foundation (@Tay_Sachs) is the only charity based in the UK dedicated to supporting those affected by #TaySachs and #Sandhoff disease.
Find out more: https://t.co/UhIcQU1h0w
The parents of the first child to receive a full dose of an experimental gene therapy for #Sandhoff disease at UMass Chan Medical School two years ago told @USATODAY they hope Alissa's short life will help others live: https://t.co/Wp1QHVpVXA #GeneTherapy #RareDisease @kweintraub
#Sandhoff disease:
🔬Lipid-storage #RareDisease
🧬lack of glucosaminidase A & B (mutations in the HEXB #gene)
🚼Typically onset < 9m: progressive psychomotor retardation
🚹Even >40y:suspected if progressive cerebellar ataxia or lower motor neuron syndrome
https://t.co/nuIyIzzDRs
✅ #SomosALAPA
👉 Cutasa es un grupo que trabaja para la cura de las enferemdades de Tay-Sachs y de Sandhoff.
📲 +54 9 11 4161-0030
#Cutasa #TaySachs #Sandhoff #Pacientes #ALAPA
Tomorrow is Rare Disease Day 2023.
This week, we will be sharing photos of some of our amazing children who live/have lived with Tay-Sachs and Sandhoff disease. We will be sharing some facts about Rare disease.
#rarediseaseday #Taysachs #Sandhoff #community #thecatsfoundation
Highlighting the importance of registries in #raredisease, @markus_ries et al. provide important data on the natural history of 8 Gangliosidoses #TaySachs #Sandhoff #GM1gangliosidosis https://t.co/uXQe1a9VIx
Publicaciones IEIM: ha sido un buen año para nuestro proyecto #CRISPR "CRISPR/nCas9-Based Genome Editing on GM2 Gangliosidoses Fibroblasts via Non-Viral Vectors" https://t.co/oH39lCcaVg @PujCiencias #RareDisease #TaySachs #Sandhoff
Daniel Lewi's journey as a parent changed dramatically when his daughter was diagnosed with Tay-Sachs, his experiences set him on the path to advocate. In this interview he shares his story and advice as a RARE Dad.
https://t.co/Dp5jP2Vgzt
#RAREParenting #taysachs #sandhoff
Watch on our #PatientEducation site: Learn about #genetherapy being researched as a potential one-time treatment for #TaySachs & #Sandhoff disease, the two most common types of #GM2 #gangliosidosis. #rarediseases https://t.co/MqrcCEHXi7
Dr Heather Gray-Edwards on drug delivery to the CNS: AAV-GM2 delivery #taysachs #sandhoff
#SNIS2022
@GuenegoAdrien @lila_sheikhi @DanniDiestro @JNIS_BMJ @SNISinfo @fcalbuquerque51 @rdeleacymd @ducruetaf @DrAltschul
@DrMichaelLevitt
@thepaulsingh @mattgounis @AjitSPuri1
Since the @Tay_Sachs was founded they been able to lead a huge rise in the research projects investigating the diseases. Although still in their infancy, they are excited by their potential to be effective at managing the symptoms of #TaySachs and #Sandhoff.
La asociación @ACTAYS se creó para encontrar cura a 2 #EnfermedadesRaras de carácter neurodegenerativo (#TaySachs y #Sandhoff) y para asistir a las familias de los niños que la padecen.
Beatriz Fernández, su fundadora, explicó su historia @OndaCero_es.
https://t.co/WNklunQ7gT
La asociación @ACTAYS se creó para encontrar cura a 2 #EnfermedadesRaras de carácter neurodegenerativo (#TaySachs y #Sandhoff) y para asistir a las familias de los niños que la padecen.
Beatriz Fernández, su fundadora, explicó su historia @OndaCero_es.
https://t.co/WNklunQ7gT
TBT #rarediseaseday2021 - a video to pay tribute to some of the many #patients that we #supported along the years and that inspire us to continue the #fight for #rarediseases.
https://t.co/fxKRtnwRzD
#rarediseaseday #taysachs #sandhoff #10years #awareness
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