Live and recent posts from across the Fediverse tagged #rettsyndrome, aggregated by home.social.
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
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
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
`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.`
https://www.europesays.com/2823359/ Acadia Faces EU Setback On Trofinetide As Valuation Signals Diverge #ACADIAPharmaceuticals #Europe #EuropeanMarket #EuropeanMedicinesAgency #IndividualInvestors #RettSyndrome
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
How Regaining Full Rights to Rett Syndrome Gene Therapy Has Changed Taysha (TSHA)’s Investment Story
Recently, Taysha Gene Therapies announced it has regained full rights to its lead gene therapy candidate TSHA-102 for…
#NewsBeep #News #Headlines #BreakthroughTherapy #clinicalsignificance #genetherapies #Latvia #LV #regulatoryapproval #regulatorysignificance #Rettsyndrome
https://www.newsbeep.com/193054/
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
Bilingualism possible in people with rare genetic condition that normally limits speech
Rett syndrome is a rare neurological condition that disrupts physical and linguistic development, affecting around one in 10,000…
#NewsBeep #News #Genetics #CA #Canada #cognitiveability #Development #developmentalcondition #EnglishandWelsh #languagedevelopment #linguisticabilities #linguisticdevelopment #researchers #Rettsyndrome #Science
https://www.newsbeep.com/ca/114625/
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/
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 [..]"
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
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
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
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.
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
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
That was a night to forget...
Asleep in the Mouse House with Graham Diering
https://www.spectrumnews.org/news/profiles/asleep-in-the-mouse-house-with-graham-diering/
#Phelan-McDermidsyndrome #Rettsyndrome #mousemodels #Profiles #synapses #autism #SHANK3 #sleep
Progress. A only woke once last night at 2 ish. She was hard to settle but after I winged my way through telling a story (just off the top of my head - couldn't risk putting the light on) she drifted off. Phew. Unfortunately I was pretty awake but I think I grabbed an hour in the end.
Morning
#LearningDisability #RettSyndrome #SETD5Syndrome #SleepDisorders
A bit niche, but I'm looking for people in the #RettSyndrome #SETD5Syndrome #Autism #LearningDisability #Epilepsy community. Our daughter has all of the above. Would love to meet fellow #ParentCarer #UnpaidCarer folks
School just phoned. A slept nearly all afternoon, then woke just in for the Wellbeing lady to give her a little aromatherapy massage before hometime. If only all schools were this flexible #RettSyndrome #SETD5Syndrome #Disability #LearningDifficulties #epilepsy
This is a beautiful piece of writing from Catriona Moore https://makingchromosomescount.co.uk/2022/11/14/just-the-way-she-is-rett-syndrome/
Shifting from doing to being - solid way for all of us to live by IMO
"Slowing down to make the world work better for Amy has made it work better for me too. I love her company and her reactions to things, and the way she lights up when something really delights her. She has shown me the value of simply being here, together".
Our daughter A, who has 2 rare syndromes and complex needs, has been living her best life out with her wonderful PA this evening #RettSyndrome #SETD5Syndrome #DisabilityMstdn #LearningDisability #Autism
How star cells’ secretions may stunt neurons: Q&A with Nicola Allen
https://www.spectrumnews.org/opinion/how-star-cells-secretions-may-stunt-neurons-qa-with-nicola-allen/
#fragileXsyndrome #Rettsyndrome #astrocytes #synapses #Opinion #autism #QA
RNA rewrite improves breathing, survival in Rett mice
https://www.spectrumnews.org/news/rna-rewrite-improves-breathing-survival-in-rett-mice/
#Rettsyndrome #genetherapy #mousemodels #autism #MECP2 #News
Portrait of a research field: astrocytes in autism
https://www.spectrumnews.org/features/deep-dive/portrait-of-a-research-field-astrocytes-in-autism/
#excitatorysignaling #inhibitorysignaling #signalingimbalance #fragileXsyndrome #postmortembrains #geneexpression #Rettsyndrome #environment #mousemodels #astrocytes #organoids #DeepDive #seizures #synapses #autism #MECP2 #FMR1 #FMRP #GABA
Faulty inhibition foils long-term memory in Rett mice
https://www.spectrumnews.org/news/faulty-inhibition-foils-long-term-memory-in-rett-mice/
#excitatorysignaling #inhibitorysignaling #learningandmemory #neuralcircuits #Rettsyndrome #hippocampus #mousemodels #learning #seizures #autism #MECP2 #News
Silenced X chromosome genes reawaken in new mouse model
https://www.spectrumnews.org/news/toolbox/silenced-x-chromosome-genes-reawaken-in-new-mouse-model/
#Rettsyndrome #mousemodels #Xchromosome #treatments #Toolbox #autism #MECP2
Protein complex points to new route to Rett syndrome
https://www.spectrumnews.org/news/protein-complex-points-to-new-route-to-rett-syndrome/
#Rettsyndrome #mousemodels #autism #MECP2 #News
Real-world data on Rett syndrome: Q&A with Jana von Hehn
https://www.spectrumnews.org/opinion/real-world-data-on-rett-syndrome-qa-with-jana-von-hehn/
#clinicaltrials #rarevariants #Rettsyndrome #treatments #Opinion #autism #MECP2 #QA
Hollis Cline: Leapfrogging over gaps in autism research
https://www.spectrumnews.org/news/profiles/hollis-cline-leapfrogging-over-gaps-in-autism-research/
#fragileXsyndrome #brainimaging #Rettsyndrome #Profiles #autism #MECP2 #FMR1