Top Tweets for #MPSIIIA
Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA - For More Information Visit https://t.co/KSTYCAxFK5 @ultragenyx #Rare_Diseases #MPSIIIA #FDA #Sanfilippo_Syndrome #Drug_Discovery
🔍 Sanfilippo tipo A en Egipto
🧬 Gen → mutaciones nuevas
👨👩👧👦 Consanguinidad >80 %
🧪 Clínica → retraso + deterioro neurológico
🌍 Impacto → registro nacional + consejería genética
@StopSanfilippo @MPSpapas
📖 https://t.co/1KzmYptG10
#MPSIIIA #EnfermedadesRaras
un #varon de 53 años #diagnosticado de síndrome de #Usher por retinitis pigmentosa y pérdida auditiva neurosensorial. El #exoma clínico , estudio enzimático y un aumento urinaria de #GAG confirmó que era #MPSIIIA @Aecom_EIM @Info_Rares @RRelagh @AEGHgenetica #EnfermedadesRaras
📢📢Huge #neurodegeneration news: @Denalitx drug (#lysosomal enzyme with brain shuttle) reduced brain damage marker NFL in lysosomal disease (#LSD) #MPSII. #MPSI, #MPSIIIA in their sights. Reminder: #Alzheimers, #Parkinsons are also lysosomal diseases.
https://t.co/uoEhkrjYan
Off to @ASH_hematology in New Orleans to present first in world gene therapy #MPSIIIA and our cord blood #transplant in refractory #Leukaemia.... Our team in @RMCH_BMTU... Love Hematology still
Two years after being dosed with an investigational #genetherapy, children with #Sanfilippo syndrome type A showed stable or continuously increasing cognitive, language, and motor functions, according to new data. https://t.co/9yrzscRtE5
Proud to share our latest work on pharmacological approach to treat the CNS pathology in #MPSIIIA
Congratulations to Antonella Capuozzo! Her latest work used cell-based high content imaging to identify #fluoxetine as a potential therapeutic to treat #MPS-IIIA by increasing lysosomal and autophagic functions via TFEB activation.
https://t.co/PlfXDMzCMw
@HCS_Tigem @cristinasorr
JCR Pharmaceuticals has received #OrphanDrug designation from the EC for their #Sanfilippo type A enzyme replacement therapy candidate JR-441. They are preparing for a global #ClinicalTrial with JR-441 in patients with #MPSIIIA to start in 2023. Read more: https://t.co/mhAEAmC6jK
Finally our "something blue" is also on treatment in MPS III and comes from the old version of JIMD Reports.
Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice
Ningning Guo et al
#MPSIIIA #mucopolysaccharidosis
https://t.co/ksfawZM2Hy
”something new” in the journal
Novel therapies for #mucopolysaccharidosis type III
Berna Seker Yilmaz, James Davison, Simon A Jones & Julien Baruteau
A recap on the historical approach up to modern gene therapy approaches in #MPSIIIA
https://t.co/YDgsaypZEQ
Free Webinar for HCPs, July 23: learn about our trials enrolling children with #MPSIIIA & #MPSIIIB. Many thanks to #MPSInternational for providing this platform in lieu of its annual meeting. Live translation into Spanish, Mandarin, & Japanese. Register: https://t.co/w8un0RDRW9
Today in our #StemCell & #GeneTherapy Seminar Series we were fortunate to have @cirmnews Bridges Student Sabrina Noritake present her project on Neural stem cells engineered to make SGSH enzyme for #SanFilippo syndrome (#MPSIIIA). Bridges is a great training program w/ @sacstate.
Best of luck to the patients, families, and healthcare teams delivering this #GeneTherapy for #SanFilippo! #MPSIIIA
1st Patient Dosed in Trial of Gene Therapy, OTL-201, for Sanfilippo Type A https://t.co/NK3VwXglUa
... predicting #AntibioticResistance, #Alzheimer ’s rose genes and #microglia (our cover, thank you
@DeStrooperLab & @skrahl), #MPSIIIA, #ChromosomeInstability, and #mitochondrialdisease
as always, #openaccess [2/2]
https://t.co/NrWHCgUNif
![EmboMolMed's tweet photo. ... predicting #AntibioticResistance, #Alzheimer ’s rose genes and #microglia (our cover, thank you
@DeStrooperLab & @skrahl), #MPSIIIA, #ChromosomeInstability, and #mitochondrialdisease
as always, #openaccess [2/2]
https://t.co/NrWHCgUNif https://t.co/6F0QQ26lUS](https://pbs.twimg.com/media/ESvFcb6XgAAxCjx.jpg)
10 days until #RareDiseaseDay. There are more than 7,000 rare diseases affecting more than 350 million people around the world. On Feb 29 our teams across the globe will unite to raise awareness of #Duchenne #MuscularDystrophy, #Limbgirdle muscular dystrophies and #MPSIIIA.
H Parker, B Bigger & coll. @OfficialUoM @WithingtonHosp report that #IL1 is a key mediator in the #mucopolysaccharidosis IIIA (#MPSIIIA) inflammatory cascade. #HematopoieticStemCell #genetherapy using IL1Ra prevented cognitive & behavioural decline in mice
https://t.co/rkXXujcKNQ
Next week, we will descend upon Orlando for the WORLDSymposium™ on Lysosomal Storage Diseases. We look forward to coming together with the community to exchange scientific knowledge helping to advance our #GeneTherapy programs. #WORLDSymposia #MPSIIIA #MPSIIIB #CLN1 #CLN3
We’re at the 33rd annual @MPSSociety Family Conference in Orlando. We look forward to hearing from families and experts, and sharing about our work to develop a potential treatment for #MPSIIIA, also known as #Sanfilippo syndrome A.
Congratulations to Cristina Sorrentino @cristinasorr and Fraldi’s group for the publication on @MolTherapy
Her work is of crucial importance for the therapeutic treatment of #MucopolysaccharidosisIIIA #MPSIIIA @CellPressNews #TigemScience
Read more here https://t.co/r7rh6iobm0
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