#rettsyndrome — Public Fediverse posts
Live and recent posts from across the Fediverse tagged #rettsyndrome, aggregated by home.social.
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European Commission Approves DAYBU® (trofinetide) as the First and Only Treatment for Neurobehavioral Symptoms of Rett Syndrome in the European Union
SAN DIEGO, August 24, 2026–(BUSINESS WIRE)–Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the European …
#Europe #EU #ACADIAPharmaceuticals #DAYBU #EuropeanCommission #EuropeanUnion #neurodevelopmentaldisorder #pediatricpatients #raredisease #Rettsyndrome #treatmentoption #treatmentoptions
https://www.europesays.com/europe/122619/ -
Brain organoid studies on the ISS move closer to new treatments
Research using human brain organoids onboard the International Space Station International Space Station (ISS) helped lead to the…
#NewsBeep #News #Health #AU #Australia #Brain #Children #clinicaltrial #Genetic #Inflammation #Laboratory #Lamivudine #multiplesclerosis #neurodegeneration #organoids #research #Rettsyndrome #Sclerosis #StemCells #Syndrome #therapy
https://www.newsbeep.com/au/862090/ -
https://www.europesays.com/ie/619219/ Advances in X chromosome inactivation open therapeutic opportunities for X-linked genetic disorders #Allele #Cell #CellBiology #Chromatin #Chromosome #DuchenneMuscularDystrophy #Éire #EmbryonicDevelopment #FabryDisease #Gene #GeneExpression #Genes #Genetic #Health #Hemophilia #IE #Ireland #MuscularDystrophy #Research #RettSyndrome #RNA #Syndrome #therapy #XChromosome #XLinked
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DAYBU® (trofinetide) Recommended for Approval in the European Union by CHMP
— European Commission decision expected in the coming months — If approved, DAYBU® would become the first treatment…
#Europe #EU #ACADIAPharmaceuticals #DAYBU #EuropeanCommission #EuropeanMedicinesAgency #EuropeanUnion #Forward-LookingStatements #marketingauthorization #Rettsyndrome #trofinetide
https://www.europesays.com/europe/80006/ -
Acadia DAYBU wins EU approval recommendation
Key Terms
committee for medicinal products for human use
regulatory
…
#Europe #EU #EuropeanCommission #ACAD #Acadia #CHMP #DAYBU #EuropeanUnion #Rettsyndrome #trofinetide
https://www.europesays.com/europe/80003/ -
https://www.europesays.com/uk/902464/ This is Why Taysha Gene Therapies, Inc. (TSHA) is one of the Best Healthcare Penny Stocks to Buy #Canaccord #GeneTherapies #Health #Healthcare #RettSyndrome #UK #UnitedKingdom
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This is Why Taysha Gene Therapies, Inc. (TSHA) is one of the Best Healthcare Penny Stocks to Buy
Taysha Gene Therapies Inc. (NASDAQ:TSHA) is one of the best healthcare penny stocks to buy according to hedge…
#NewsBeep #News #Healthcare #AU #Australia #Canaccord #genetherapies #Health #Rettsyndrome
https://www.newsbeep.com/au/614197/ -
This is Why Taysha Gene Therapies, Inc. (TSHA) is one of the Best Healthcare Penny Stocks to Buy
Taysha Gene Therapies Inc. (NASDAQ:TSHA) is one of the best healthcare penny stocks to buy according to hedge…
#NewsBeep #News #Healthcare #Canaccord #genetherapies #Health #healthcare #Rettsyndrome #UK #UnitedKingdom
https://www.newsbeep.com/uk/535221/ -
https://www.europesays.com/ie/439221/ This is Why Taysha Gene Therapies, Inc. (TSHA) is one of the Best Healthcare Penny Stocks to Buy #Canaccord #Éire #GeneTherapies #Health #HealthCare #Healthcare #IE #Ireland #RettSyndrome
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`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`
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`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`
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`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`
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`These data set the stage for a potential therapeutic strategy using antisense oligonucleotides to promote isoform switching in patients with RTT who carry partially functioning alleles of MECP2.`
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https://www.instagram.com/reel/DVGWNjKDPJk/?igsh=MWZmbTFqaG9zNXdjaQ==
Handiski 2026 is not a “nice activity”.
It’s dignity in motion.In February, 6 families.
6 girls with Rett syndrome.
3 days in La Bresse.
Full inclusion. Full access. Zero financial barrier.Ski sessions adapted.
Siblings in ski school.
Parents breathing for once.This is what inclusion looks like when you stop talking and start funding.
Support the mission 👉 https://alsr.lu
#Handiski #RettSyndrome #Inclusion #DisabilityRights #ALSR -
Handiski 2026 is not a “charity trip.”
It’s training.
It’s courage.
It’s trust.Five girls.
Adaptive equipment.
Cold mornings.
Zero excuses.When you see a wheelchair on snow, you don’t see limits.
You see logistics, teamwork, and pure determination.We’re preparing.
#Handiski2026 #RettSyndrome #AdaptiveSport #Inclusion -
Handiski 2026 is officially loading ❄️
After La Bresse 2025, we’re going back to the mountains with the girls of ALSR.
Skiing. Laughter. Cold air. Real freedom.For girls living with Rett syndrome, this isn’t “just” a trip.
It’s mobility. Inclusion. Dignity. Joy.More soon.
#Handiski2026 #RettSyndrome #Inclusion #Luxembourg -
https://www.europesays.com/uk/392412/ Bilingualism possible in people with rare genetic condition that normally limits speech #CognitiveAbility #Development #DevelopmentalCondition #EnglishAndWelsh #Genetics #LanguageDevelopment #LinguisticAbilities #LinguisticDevelopment #researchers #RettSyndrome #Science #UK #UnitedKingdom
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Brilliant work by #WhiteheadInstitute researchers and collaborators unpacking MECP2’s complex role in Rett syndrome - as both a gene activator and repressor. A powerful step toward more targeted therapies. #RettSyndrome #Neuroscience #Genetics #MECP2 #ScienceMatters #JaenischLab #YoungLab
RE: https://bsky.app/profile/did:plc:376brirdwae4fayb4sd3bzdy/post/3lp5hlxwl722j -
Brilliant work by #WhiteheadInstitute researchers and collaborators unpacking MECP2’s complex role in Rett syndrome - as both a gene activator and repressor. A powerful step toward more targeted therapies. #RettSyndrome #Neuroscience #Genetics #MECP2 #ScienceMatters #JaenischLab #YoungLab
RE: https://bsky.app/profile/did:plc:376brirdwae4fayb4sd3bzdy/post/3lp5hlxwl722j -
Brilliant work by #WhiteheadInstitute researchers and collaborators unpacking MECP2’s complex role in Rett syndrome - as both a gene activator and repressor. A powerful step toward more targeted therapies. #RettSyndrome #Neuroscience #Genetics #MECP2 #ScienceMatters #JaenischLab #YoungLab
RE: https://bsky.app/profile/did:plc:376brirdwae4fayb4sd3bzdy/post/3lp5hlxwl722j -
New Study Uncovers the Hidden #Genetic Cascade Behind #RettSyndrome
https://scitechdaily.com/new-study-uncovers-the-hidden-genetic-cascade-behind-rett-syndrome/
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New Study Uncovers the Hidden #Genetic Cascade Behind #RettSyndrome
https://scitechdaily.com/new-study-uncovers-the-hidden-genetic-cascade-behind-rett-syndrome/
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New Study Uncovers the Hidden #Genetic Cascade Behind #RettSyndrome
https://scitechdaily.com/new-study-uncovers-the-hidden-genetic-cascade-behind-rett-syndrome/
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new paper: "Drug repurposing in Rett and Rett-like syndromes: a promising yet underrated opportunity?" https://doi.org/10.3389/fmed.2024.1425038
"Rett syndrome (RTT) and Rett-like syndromes [i.e., CDKL5 deficiency disorder (CDD) and FOXG1-syndrome] represent rare yet profoundly impactful neurodevelopmental disorders (NDDs). [..] The current landscape of DR efforts in these syndromes is thoroughly examined [..]"
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new paper: "Drug repurposing in Rett and Rett-like syndromes: a promising yet underrated opportunity?" https://doi.org/10.3389/fmed.2024.1425038
"Rett syndrome (RTT) and Rett-like syndromes [i.e., CDKL5 deficiency disorder (CDD) and FOXG1-syndrome] represent rare yet profoundly impactful neurodevelopmental disorders (NDDs). [..] The current landscape of DR efforts in these syndromes is thoroughly examined [..]"
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new paper: "Drug repurposing in Rett and Rett-like syndromes: a promising yet underrated opportunity?" https://doi.org/10.3389/fmed.2024.1425038
"Rett syndrome (RTT) and Rett-like syndromes [i.e., CDKL5 deficiency disorder (CDD) and FOXG1-syndrome] represent rare yet profoundly impactful neurodevelopmental disorders (NDDs). [..] The current landscape of DR efforts in these syndromes is thoroughly examined [..]"
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It seems that a genetic mutation may not have the same physiological effect on different types of cells.
"This shows that in order to understand Rett syndrome, we need to look beyond what's happening in neurons to other cell types," said first study author Danielle Tomasello, PhD.
#genetics #mutation #biology #research #health #medicine #disease #RettSyndrome
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It seems that a genetic mutation may not have the same physiological effect on different types of cells.
"This shows that in order to understand Rett syndrome, we need to look beyond what's happening in neurons to other cell types," said first study author Danielle Tomasello, PhD.
#genetics #mutation #biology #research #health #medicine #disease #RettSyndrome
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It seems that a genetic mutation may not have the same physiological effect on different types of cells.
"This shows that in order to understand Rett syndrome, we need to look beyond what's happening in neurons to other cell types," said first study author Danielle Tomasello, PhD.
#genetics #mutation #biology #research #health #medicine #disease #RettSyndrome
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It seems that a genetic mutation may not have the same physiological effect on different types of cells.
"This shows that in order to understand Rett syndrome, we need to look beyond what's happening in neurons to other cell types," said first study author Danielle Tomasello, PhD.
#genetics #mutation #biology #research #health #medicine #disease #RettSyndrome
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Angry mum says disabled daughter has 'shut down' after water damage left her without bed - Mirror Online
https://www.mirror.co.uk/news/uk-news/angry-mum-says-disabled-daughter-31977516#Disabilities
#CareHomes
#RettSyndrome
#Glasgow
#HaydaleCareHome
#Scotland -
Angry mum says disabled daughter has 'shut down' after water damage left her without bed - Mirror Online
https://www.mirror.co.uk/news/uk-news/angry-mum-says-disabled-daughter-31977516#Disabilities
#CareHomes
#RettSyndrome
#Glasgow
#HaydaleCareHome
#Scotland -
Angry mum says disabled daughter has 'shut down' after water damage left her without bed - Mirror Online
https://www.mirror.co.uk/news/uk-news/angry-mum-says-disabled-daughter-31977516#Disabilities
#CareHomes
#RettSyndrome
#Glasgow
#HaydaleCareHome
#Scotland -
Angry mum says disabled daughter has 'shut down' after water damage left her without bed - Mirror Online
https://www.mirror.co.uk/news/uk-news/angry-mum-says-disabled-daughter-31977516#Disabilities
#CareHomes
#RettSyndrome
#Glasgow
#HaydaleCareHome
#Scotland -
Multisite clinical trial will compare three FDA-approved drugs including ketamine for Rett syndrome treatment - SCIENMAG https://apple.news/AlQ_hWGe6Q-eyORPwzFZx_A #ketamine #psychedelic #Rettsyndrome
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Multisite clinical trial will compare three FDA-approved drugs including ketamine for Rett syndrome treatment - SCIENMAG https://apple.news/AlQ_hWGe6Q-eyORPwzFZx_A #ketamine #psychedelic #Rettsyndrome
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Genetic and Protein Network Underlying the Convergence of Rett-Syndrome-like (RTT-L) Phenotype in Neurodevelopmental Disorders. https://doi.org/10.3390/cells12101437 #RettSyndrome #AtypicalRttSyndrome #RettSyndromeLikePhenotype #MethylCpgBindingProtein2 #NeurodevelopmentalDisorders
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Genetic and Protein Network Underlying the Convergence of Rett-Syndrome-like (RTT-L) Phenotype in Neurodevelopmental Disorders. https://doi.org/10.3390/cells12101437 #RettSyndrome #AtypicalRttSyndrome #RettSyndromeLikePhenotype #MethylCpgBindingProtein2 #NeurodevelopmentalDisorders
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Genetic and Protein Network Underlying the Convergence of Rett-Syndrome-like (RTT-L) Phenotype in Neurodevelopmental Disorders. https://doi.org/10.3390/cells12101437 #RettSyndrome #AtypicalRttSyndrome #RettSyndromeLikePhenotype #MethylCpgBindingProtein2 #NeurodevelopmentalDisorders
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Genetic and Protein Network Underlying the Convergence of Rett-Syndrome-like (RTT-L) Phenotype in Neurodevelopmental Disorders. https://doi.org/10.3390/cells12101437 #RettSyndrome #AtypicalRttSyndrome #RettSyndromeLikePhenotype #MethylCpgBindingProtein2 #NeurodevelopmentalDisorders
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FDA approval of trofinetide may spur further drug development for Rett
https://www.spectrumnews.org/news/fda-approval-of-trofinetide-may-spur-further-drug-development-for-rett/
#Rettsyndrome #mousemodels #treatments #autism #MECP2 #News -
FDA approval of trofinetide may spur further drug development for Rett
https://www.spectrumnews.org/news/fda-approval-of-trofinetide-may-spur-further-drug-development-for-rett/
#Rettsyndrome #mousemodels #treatments #autism #MECP2 #News -
FDA approval of trofinetide may spur further drug development for Rett
https://www.spectrumnews.org/news/fda-approval-of-trofinetide-may-spur-further-drug-development-for-rett/
#Rettsyndrome #mousemodels #treatments #autism #MECP2 #News -
Happy #MedicineMonday! The #FDA just approved the first treatment for #RettSyndrome, a rare, genetic, neurological disorder affecting brain development. This condition affects about 1:10,000 females, males to a far rarer extent and leads to a loss of language and motor skills developed earlier in life.
The medication Daybue (trofinetide) is for people 2 or older, delivered orally or via gastrostomy tube.
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Happy #MedicineMonday! The #FDA just approved the first treatment for #RettSyndrome, a rare, genetic, neurological disorder affecting brain development. This condition affects about 1:10,000 females, males to a far rarer extent and leads to a loss of language and motor skills developed earlier in life.
The medication Daybue (trofinetide) is for people 2 or older, delivered orally or via gastrostomy tube.
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Happy #MedicineMonday! The #FDA just approved the first treatment for #RettSyndrome, a rare, genetic, neurological disorder affecting brain development. This condition affects about 1:10,000 females, males to a far rarer extent and leads to a loss of language and motor skills developed earlier in life.
The medication Daybue (trofinetide) is for people 2 or older, delivered orally or via gastrostomy tube.
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Happy #MedicineMonday! The #FDA just approved the first treatment for #RettSyndrome, a rare, genetic, neurological disorder affecting brain development. This condition affects about 1:10,000 females, males to a far rarer extent and leads to a loss of language and motor skills developed earlier in life.
The medication Daybue (trofinetide) is for people 2 or older, delivered orally or via gastrostomy tube.
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Debut drug for Rett syndrome at edge of approval
https://www.spectrumnews.org/news/debut-drug-for-rett-syndrome-at-edge-of-approval/
#fragileXsyndrome #Rettsyndrome #mousemodels #treatments #autism #DSM-IV #MECP2 #News -
Debut drug for Rett syndrome at edge of approval
https://www.spectrumnews.org/news/debut-drug-for-rett-syndrome-at-edge-of-approval/
#fragileXsyndrome #Rettsyndrome #mousemodels #treatments #autism #DSM-IV #MECP2 #News -
Going on Trial: Orphan drugs; CBD; bumetanide
https://www.spectrumnews.org/news/going-on-trial-orphan-drugs-cbd-bumetanide/
#inhibitorysignaling #fragileXsyndrome #clinicaltrials #Rettsyndrome #genetherapy #mousemodels #funding #autism #22q11 #MECP2 #News #FMR1 -
Going on Trial: Orphan drugs; CBD; bumetanide
https://www.spectrumnews.org/news/going-on-trial-orphan-drugs-cbd-bumetanide/
#inhibitorysignaling #fragileXsyndrome #clinicaltrials #Rettsyndrome #genetherapy #mousemodels #funding #autism #22q11 #MECP2 #News #FMR1 -
Going on Trial: Orphan drugs; CBD; bumetanide
https://www.spectrumnews.org/news/going-on-trial-orphan-drugs-cbd-bumetanide/
#inhibitorysignaling #fragileXsyndrome #clinicaltrials #Rettsyndrome #genetherapy #mousemodels #funding #autism #22q11 #MECP2 #News #FMR1 -
Biotech downturn hurts companies targeting autism-linked conditions
https://www.spectrumnews.org/news/biotech-downturn-hurts-companies-targeting-autism-linked-conditions/
#clinicaltrials #Rettsyndrome #funding #autism #News -
Biotech downturn hurts companies targeting autism-linked conditions
https://www.spectrumnews.org/news/biotech-downturn-hurts-companies-targeting-autism-linked-conditions/
#clinicaltrials #Rettsyndrome #funding #autism #News -
Biotech downturn hurts companies targeting autism-linked conditions
https://www.spectrumnews.org/news/biotech-downturn-hurts-companies-targeting-autism-linked-conditions/
#clinicaltrials #Rettsyndrome #funding #autism #News -
Epigenome edits unmute MECP2 in Rett-like neurons
https://www.spectrumnews.org/news/epigenome-edits-unmute-mecp2-in-rett-like-neurons/
#Rettsyndrome #genetherapy #mousemodels #autism #MECP2 #News -
Epigenome edits unmute MECP2 in Rett-like neurons
https://www.spectrumnews.org/news/epigenome-edits-unmute-mecp2-in-rett-like-neurons/
#Rettsyndrome #genetherapy #mousemodels #autism #MECP2 #News -
Epigenome edits unmute MECP2 in Rett-like neurons
https://www.spectrumnews.org/news/epigenome-edits-unmute-mecp2-in-rett-like-neurons/
#Rettsyndrome #genetherapy #mousemodels #autism #MECP2 #News -
That was a night to forget...
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That was a night to forget...