#raredisease — Public Fediverse posts

A New RNA Therapy Study Is Changing How Scientists Think About Severe Childhood Epilepsy https://www.growingyourbaby.com/could-rare-epilepsy-treatment-begin-before-birth/ #ParentingNews #ChildHealth #RareDisease #EpilepsyAwareness #GeneticResearch #PregnancyResearch #MedicalResearch #research

A New RNA Therapy Study Is Changing How Scientists Think About Severe Childhood Epilepsy https://www.growingyourbaby.com/could-rare-epilepsy-treatment-begin-before-birth/ #ParentingNews #ChildHealth #RareDisease #EpilepsyAwareness #GeneticResearch #PregnancyResearch #MedicalResearch #research

A New RNA Therapy Study Is Changing How Scientists Think About Severe Childhood Epilepsy https://www.growingyourbaby.com/could-rare-epilepsy-treatment-begin-before-birth/ #ParentingNews #ChildHealth #RareDisease #EpilepsyAwareness #GeneticResearch #PregnancyResearch #MedicalResearch #research

A New RNA Therapy Study Is Changing How Scientists Think About Severe Childhood Epilepsy https://www.growingyourbaby.com/could-rare-epilepsy-treatment-begin-before-birth/ #ParentingNews #ChildHealth #RareDisease #EpilepsyAwareness #GeneticResearch #PregnancyResearch #MedicalResearch #research

https://www.europesays.com/dk/84076/ Novo Nordisk Abandons Rare-Disease Factory for Warehouse Push as Canada Opens Door to Semaglutid Gen #Abandons #Canada #door #factory #Nordisk #novo #NovoNordisk #Opens #Push #RareDisease #Warehouse

https://www.europesays.com/dk/83945/ Novo Nordisk Scraps Rare-Disease Plant and Unleashes High-Dose Data to Fortify GLP-1 Empire #data #Fortify #GLP1 #HighDose #Nordisk #novo #NovoNordisk #plant #RareDisease #Scraps #Unleashes

Soligenix advances HyBryte, a visible light photodynamic therapy for rare cutaneous T-cell lymphoma, with Phase 3 interim results expected early 2026. Novel approach offers faster response and favorable safety profile. #RareDisease #Biotech

Soligenix advances HyBryte, a visible light photodynamic therapy for rare cutaneous T-cell lymphoma, with Phase 3 interim results expected early 2026. Novel approach offers faster response and favorable safety profile. #RareDisease #Biotech

Soligenix advances HyBryte, a visible light photodynamic therapy for rare cutaneous T-cell lymphoma, with Phase 3 interim results expected early 2026. Novel approach offers faster response and favorable safety profile. #RareDisease #Biotech

Soligenix advances HyBryte, a visible light photodynamic therapy for rare cutaneous T-cell lymphoma, with Phase 3 interim results expected early 2026. Novel approach offers faster response and favorable safety profile. #RareDisease #Biotech

Soligenix advances HyBryte, a visible light photodynamic therapy for rare cutaneous T-cell lymphoma, with Phase 3 interim results expected early 2026. Novel approach offers faster response and favorable safety profile. #RareDisease #Biotech

Soligenix receives UK Promising Innovative Medicine designation for SGX945 (dusquetide) in Behçet's disease treatment, advancing its rare disease pipeline. #RareDisease #Biotech

Soligenix receives UK Promising Innovative Medicine designation for SGX945 (dusquetide) in Behçet's disease treatment, advancing its rare disease pipeline. #RareDisease #Biotech

Soligenix receives UK Promising Innovative Medicine designation for SGX945 (dusquetide) in Behçet's disease treatment, advancing its rare disease pipeline. #RareDisease #Biotech

Soligenix receives UK Promising Innovative Medicine designation for SGX945 (dusquetide) in Behçet's disease treatment, advancing its rare disease pipeline. #RareDisease #Biotech

Soligenix receives UK Promising Innovative Medicine designation for SGX945 (dusquetide) in Behçet's disease treatment, advancing its rare disease pipeline. #RareDisease #Biotech

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating the company's rare disease pipeline strategy. #Biotech #RareDisease

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating the company's rare disease pipeline strategy. #Biotech #RareDisease

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating the company's rare disease pipeline strategy. #Biotech #RareDisease

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating the company's rare disease pipeline strategy. #Biotech #RareDisease

Soligenix receives European Commission orphan drug designation for SGX945 in Behçet's disease treatment, validating the company's rare disease pipeline strategy. #Biotech #RareDisease

https://www.europesays.com/dk/82796/ Novo Nordisk Scraps Rare-Disease Factory, Bets on Oral Obesity Pill and CagriSema to Revive Growth #Bets #cagrisema #factory #Nordisk #novo #NovoNordisk #Obesity #Oral #pill #RareDisease #Scraps

France and Netherlands say all hantavirus contact cases negative

PARIS — France’s health minister s…
#Netherlands #Nederland #NL #Europe #Europa #EU #Amsterdam #closecontacts #Dutchauthorities #EUcoordination #EuropeanUnion #France #Francehealthminister #Hantavirus #healthprotocols #hospitalisolation #internationalalert #Johannesburg #MVHondiuscruiseship #precautionarymeasure #publichealth #raredisease #rodenttransmission #SaintHelena #StephanieRist
https://www.europesays.com/netherlands/11528/

Base editing approach repairs deadly Dravet syndrome mutation in mice

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially…
#NewsBeep #News #Health #Adenine #AU #Australia #Brain #Children #dna #DravetSyndrome #Epilepsy #food #Gene #Genetic #Hospital #Laboratory #MedicalSchool #medicine #MouseModel #Mutation #Neurons #Preclinical #raredisease #research #Syndrome
https://www.newsbeep.com/au/669882/

Base editing approach repairs deadly Dravet syndrome mutation in mice

Gene editing can repair a DNA error in mice that causes Dravet syndrome, a rare, incurable, and potentially…
#NewsBeep #News #Health #Adenine #AU #Australia #Brain #Children #dna #DravetSyndrome #Epilepsy #food #Gene #Genetic #Hospital #Laboratory #MedicalSchool #medicine #MouseModel #Mutation #Neurons #Preclinical #raredisease #research #Syndrome
https://www.newsbeep.com/au/669882/

Pipeline release! nf-core/raredisease v3.0.0 - 3.0.0 - Mario!
Call and score variants from WGS/WES of rare disease patients.
Please see the changelog: https://github.com/nf-core/raredisease/releases/tag/3.0.0

#diagnostics #raredisease #snv #structuralvariants #variantannotation #variantcalling #wes #wgs #nfcore #openscience #nextflow #bioinformatics

Pipeline release! nf-core/raredisease v3.0.0 - 3.0.0 - Mario!
Call and score variants from WGS/WES of rare disease patients.
Please see the changelog: https://github.com/nf-core/raredisease/releases/tag/3.0.0

#diagnostics #raredisease #snv #structuralvariants #variantannotation #variantcalling #wes #wgs #nfcore #openscience #nextflow #bioinformatics

Pipeline release! nf-core/raredisease v3.0.0 - 3.0.0 - Mario!
Call and score variants from WGS/WES of rare disease patients.
Please see the changelog: https://github.com/nf-core/raredisease/releases/tag/3.0.0

#diagnostics #raredisease #snv #structuralvariants #variantannotation #variantcalling #wes #wgs #nfcore #openscience #nextflow #bioinformatics

Pipeline release! nf-core/raredisease v3.0.0 - 3.0.0 - Mario!
Call and score variants from WGS/WES of rare disease patients.
Please see the changelog: https://github.com/nf-core/raredisease/releases/tag/3.0.0

#diagnostics #raredisease #snv #structuralvariants #variantannotation #variantcalling #wes #wgs #nfcore #openscience #nextflow #bioinformatics

Pipeline release! nf-core/raredisease v3.0.0 - 3.0.0 - Mario!
Call and score variants from WGS/WES of rare disease patients.
Please see the changelog: https://github.com/nf-core/raredisease/releases/tag/3.0.0

#diagnostics #raredisease #snv #structuralvariants #variantannotation #variantcalling #wes #wgs #nfcore #openscience #nextflow #bioinformatics

Liebe Mitmenschen.
Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft!
#Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr!

Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Liebe Mitmenschen.
Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft!
#Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr!

Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Liebe Mitmenschen.
Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft!
#Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr!

Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Liebe Mitmenschen.
Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft!
#Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr!

Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Liebe Mitmenschen.
Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft!
#Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr!

Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft! #Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr! Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft! #Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr! Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft! #Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr! Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft! #Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr! Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

Nur noch bis Ende 11.05.2026! #icd11jetzt - Schnellere & bessere Hilfe für Kranke. Bitte zeichnet!
JEDE Stimme hilft! #Lyme #mecfs #LongCovid #lgbtq+ #ChronicPain #RareDisease und viele mehr! Petitions-Link:
https://epetitionen.bundestag.de/content/petitionen/_2025/_09/_16/Petition_186702.html
.

FDA turmoil casts shadow over gene therapy conference in Europe

Andrew Joseph covers health, medicine, and the biopharma industry in Europe. You can reach Andrew on Signal at…
#Europe #EU #drugdevelopment #FDA #policy #raredisease #STAT+
https://www.europesays.com/europe/32957/

Rhythm gets EU approval for IMCIVREE in rare obesity

Rhythm Pharmaceuticals (NASDAQ: RYTM) announced the European Commission authorized IMCIVREE (setmelanotide) for treatment of obesity and control of…
#Europe #EU #acquiredhypothalamicobesity #EuropeanCommissionauthorization #EuropeanUnion #IMCIVREE #obesitytreatment #Phase3TRANSCEND #raredisease #RhythmPharmaceuticals #RYTM
https://www.europesays.com/europe/28270/

https://www.europesays.com/dk/72346/ Novo Nordisk Broadens GLP 1 Story With Pediatric And Rare Disease Gains #IndividualInvestors #NovoNordisk #ObesityAndDiabetes #RareDisease #SickleCellDisease #Type2Diabetes

https://www.europesays.com/dk/69812/ Novo Nordisk Rare Disease Wins Add Context To Undervalued Share Price #FastTrackDesignation #NovoNordisk #RareDisease #SickleCellDisease

Crinetics wins EU approval for acromegaly drug Palsonify

Crinetics Pharmaceuticals (NASDAQ: CRNX) announced European Commission approval of PALSONIFY (paltusotine) for treatment…
#Europe #EU #EuropeanCommission #acromegaly #CrineticsPharmaceuticals #CRNX #drugapproval #EuropeanCommissionapproval #Palsonify #paltusotine #raredisease
https://www.europesays.com/europe/24538/

Fatal Familial Insomnia is a rare inherited prion disease that progressively destroys the brain’s ability to regulate sleep and autonomic function. Research is now exploring gene-targeted therapies.

#FatalFamilialInsomnia #RareDisease #PrionDisease #Neurology #Telehealth

Fatal Familial Insomnia is a rare inherited prion disease that progressively destroys the brain’s ability to regulate sleep and autonomic function. Research is now exploring gene-targeted therapies.

#FatalFamilialInsomnia #RareDisease #PrionDisease #Neurology #Telehealth

Fatal Familial Insomnia is a rare inherited prion disease that progressively destroys the brain’s ability to regulate sleep and autonomic function. Research is now exploring gene-targeted therapies.

#FatalFamilialInsomnia #RareDisease #PrionDisease #Neurology #Telehealth

Fatal Familial Insomnia is a rare inherited prion disease that progressively destroys the brain’s ability to regulate sleep and autonomic function. Research is now exploring gene-targeted therapies.

#FatalFamilialInsomnia #RareDisease #PrionDisease #Neurology #Telehealth

https://www.europesays.com/ch/?p=53795 Biogen upgraded by UBS on ‘slew of pipeline catalysts’ going into 2027 #Alzheimer'sDisease #Biogen #ClinicalData #CommercialCatalysts #RareDisease #UBS