Top Tweets for #FABRY
#RAREVIEW26 nos trajo lo último en #EnfermedadesRaras #EnfermedadesLisosomales.
¡Eso incluyó también los posters presentados en San Diego sobre #Fabry, #Gaucher, #Pompe, #ASMD, #MPS y más!
Descubre en Campus Sanofi los pósters del 22nd #WORLDSymposium y consulta los hallazgos de los autores.
Descargables en formato PDF y ordenados por patología.
https://t.co/CURyyOFRZ3
🔬 Si eres profesional sanitario y tratas pacientes con enfermedad de #Fabry, puedes unirte a #FabryLink, grupo privado de #LinkedIn para conectar con expertos 🧩
Únete aquí 👉 https://t.co/3xCVrxE5D1
#EnfermedaddeFabry #RedFabry
Por cierto, ¿eres profesional sanitario y todavía no conoces #Fabrylink?
Encuentra en LinkedIn este grupo de expertos en #Fabry #EnfermedadDeFabry obtener información o compartir conocimientos. #RedFabry
https://t.co/D4mC8DyHj6
🧬 En mujeres con enfermedad de #Fabry, la actividad enzimática puede ser normal pese a existir síntomas y daño orgánico. El diagnóstico requiere integración genética + biomarcadores como Lyso-Gb3, clave para detectar la enfermedad antes de daño irreversible. 🧪💜 #Fabrylink 👇
Esperamos que os guste el artículo!
@olayahuergo_ @Sciencedirect @MedClinBarc
#Fabry #MedicinaInterna #EnfermedadesRaras
#RAREVIEW26 nos trajo lo último en #EnfermedadesRaras #EnfermedadesLisosomales.
¡Eso incluyó también los posters presentados en San Diego sobre #Fabry, #Gaucher, #Pompe, #ASMD, #MPS y más!
Descubre en Campus Sanofi los pósters del 22nd #WORLDSymposium y consulta los hallazgos de los autores.
Descargables en formato PDF y ordenados por patología.
https://t.co/CURyyOFRZ3
Cantor Fitzgerald raises its @PTCBio price target again - from $130 to $137, maintaining Overweight - explicitly citing the completed ST-920/#Fabry deal.
That’s ~110% upside from yesterday’s $65.26 close.
$PTCT $SGMO $SGMOQ @Official_Cantor
https://t.co/Rk1uJuyKFz
Thank you to everyone who visited our booth at the National Fabry Disease Foundation’s Annual Family Conference this weekend. It was inspiring to engage with the #Fabry community and see such strong interest in the future of #genetherapy.
¿Eres profesional sanitario vinculado al abordaje de #EnfermedadesRaras?
Campus Sanofi ofrece contenido especializado para especialistas, pero también para sus pacientes.
Descubre aquí, por ejemplo, la guía descargable “Vivir con #Fabry: tomando el control de cada aspecto de tu vida”
https://t.co/46UwBoLWGl
Big update for $PTCT / $SGMO $SGMOQ:
At Morgan Stanley, PTC CEO Matthew Klein on the ST-920 #Fabry deal:
“The deal should close in the next week or so.”
BLA completion targeted by year-end, with a potential 2027 launch. 🚀
@PTCBio @SangamoTx
We will be attending and presenting at this week’s Fabry Family Education Conference in Greensboro, NC. We look forward to participating and connecting with the #Fabry community.
En #Fabry, el corazón se convierte en un órgano silenciosamente afectado.
¿Eres profesional sanitario? ¿Quieres saber más sobre esta enfermedad de depósito lisosomal?
#FabryLink es apoyo e información sobre la #EnfermedadDeFabry + conexión con otros expertos nacionales e internacionales en un grupo de LinkedIn.
Entra aquí para formar parte:
https://t.co/ZlOqNdpbUh
AMT-191 gene therapy in males with Fabry disease; phase 1/2 initial safety and biomarker results
https://t.co/alfzHMt0Sr
$QURE #Fabry
https://t.co/HxXB2GqIRm
In late August, our team participated at SSIEM 2026 in Helsinki, Finland where uniQure’s Ari Pano and Christy Quintana presented updates on safety data related to our #Fabry #genetherapy trial. It was a great opportunity to connect with the community and discuss the path forward.
In late August, our team participated at SSIEM 2026 in Helsinki, Finland where uniQure’s Ari Pano and Christy Quintana presented updates on safety data related to our #Fabry #genetherapy trial. It was a great opportunity to connect with the community and discuss the path forward.
🧬 #SSIEM2026 | Post 2
Exciting preliminary data on AMT-191 #genetherapy for #Fabry disease:
🔹 Sustained α-GAL activity after a single dose
🔹 11 patients maintained enzyme activity after stopping ERT
🔹 Lyso-Gb3 remained stable or decreased in several patients
🔹 Safety was generally manageable, although 3 grade-3 liver DLTs require close monitoring.
A promising step towards durable, potentially ERT-free treatment for Fabry disease. 🚀
$SGMO speaking today and tomorrow:
#FABRY #Accelerated #BLA
late-stage AAV program for #accelerated #CMC development in support of the approval pathway.
A case study and some #data will be presented
#MINT genome-editing therapeutics and the key considerations required ..
Más allá de #Gaucher, #Fabry, #Pompe, #ASMD, #MPS1…
Los Drs. Jorge Gómez y Javier de las Heras repasaron en #RAREVIEW26 las novedades presentadas en el #WORLDSymposium2026 sobre otras #EnfermedadesLisosomales.
Con foco en Niemann-Pick tipo C y gangliosidosis GM1/GM2.
https://t.co/9Oi4KtFxF8
People with #Fabry disease face a significantly increased risk of stroke because the disease progressively damages blood vessels and the heart. As a result, strokes can occur at a relatively young age, even in the absence of traditional cardiovascular risk factors. The article highlights the importance of regular monitoring and comprehensive disease management. This is where $SGMO s ST-920 (isaralgagene civaparvovec) could offer a meaningful long-term advantage: by providing sustained α-galactosidase A expression and addressing the underlying cause of vascular injury, the gene therapy has the potential to reduce future stroke risk. Whether this potential translates into improved long-term clinical outcomes remains to be confirmed through extended follow-up.
https://t.co/BVrlCeU9YI
"Antibodies to Fabry treatment tied to infusion reaction risk"
$SGMO
The study is particularly interesting because it highlights another potential drawback of lifelong enzyme replacement therapy in #Fabry disease: some patients develop antibodies against the infused enzyme. These anti-drug antibodies can be associated with higher disease markers such as Lyso-Gb3 and, in particular, an increased risk of infusion-related reactions. However, a clear link to faster kidney or heart damage has not yet been established. For ST-920, this is nevertheless relevant: if the one-time gene therapy successfully enables the body to produce α-galactosidase A itself, it could eliminate the need for lifelong, biweekly enzyme infusions and potentially avoid the associated ADA problem. The benefit is not completely immunologically “free,” however, as AAV gene therapy can itself trigger immune responses against the vector.
https://t.co/wTP0bRMKpw
#RAREVIEW26 trajo de nuevo más lo más destacado en #EnfermedadesLisosomales del #WorldSymposium2026 de San Diego.
¿No pudiste asistir?
💻 Ya están disponibles las grabaciones de la jornada, con lo último en #Fabry, #Gaucher, #Pompe, #ASMD, #MPS y más enfermedades raras.
🥼 Contenido para profesionales sanitarios.
👉https://t.co/mQmx6rr1zQ
Last Seen Hashtags on Sotwe
Most Popular Users
Elon Musk 
@elonmusk
241.8M followers
Barack Obama
@barackobama
119M followers
Cristiano Ronaldo
@cristiano
114.6M followers
Donald J. Trump
@realdonaldtrump
111.9M followers
Narendra Modi
@narendramodi
107.2M followers
Rihanna
@rihanna
98.7M followers
NASA
@nasa
92.4M followers
Justin Bieber
@justinbieber
91.8M followers
KATY PERRY
@katyperry
90.1M followers
Taylor Swift
@taylorswift13
84M followers
Lady Gaga
@ladygaga
75.5M followers
Virat Kohli
@imvkohli
73.5M followers
Kim Kardashian
@kimkardashian
70.9M followers
YouTube
@youtube
68.8M followers
Neymar Jr
@neymarjr
66.5M followers
Bill Gates
@billgates
65.3M followers
Selena Gomez
@selenagomez
63.1M followers
The Ellen Show
@theellenshow
62.3M followers
CNN
@cnn
61.8M followers
X
@x
60.7M followers