#duchennemusculardystrophy — Public Fediverse posts
Live and recent posts from across the Fediverse tagged #duchennemusculardystrophy, aggregated by home.social.
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https://www.europesays.com/ch/97327/ Novartis delpacibart braxlosiran (del-brax) Phase I/II study in facioscapulohumeral muscular dystrophy (FSHD) meets primary biomarker endpoint #ClinicalStudies #DuchenneMuscularDystrophy #FSHD #MuscleDamage #MuscularDystrophy #NeuromuscularDisease #Novartis #patients #PhaseIII #PhaseIIITrial #SpinalMuscularAtrophy
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Designer featured on Gardening Australia stripped of NDIS support funding
A Central Victorian man says the National Disability Insurance Agency (NDIA) has robbed him of dignity and is…
#Australia #AU #Austrlia #DuchenneMuscularDystrophy #garden #gardeningaustralia #joshuaruff #nationaldisabilityinsurancescheme #ndia #wheelchair
https://www.europesays.com/australia/40371/ -
Designer featured on Gardening Australia stripped of NDIS support funding
A Central Victorian man says the National Disability Insurance Agency (NDIA) has robbed him of dignity and is…
#NewsBeep #News #Sports #AU #Australia #duchennemusculardystrophy #Garden #GardeningAustralia #joshuaruff #nationaldisabilityinsurancescheme #ndia #sports #Wheelchair
https://www.newsbeep.com/au/766710/ -
Designer featured on Gardening Australia stripped of NDIS support funding
A Central Victorian man says the National Disability Insurance Agency (NDIA) has robbed him of dignity and is…
#NewsBeep #News #Sports #AU #Australia #duchennemusculardystrophy #Garden #GardeningAustralia #joshuaruff #nationaldisabilityinsurancescheme #ndia #sports #Wheelchair
https://www.newsbeep.com/au/766710/ -
Designer featured on Gardening Australia stripped of NDIS support funding https://www.allforgardening.com/1846147/designer-featured-on-gardening-australia-stripped-of-ndis-support-funding/ #DuchenneMuscularDystrophy #garden #gardening #GardeningAustralia #JoshuaRuff #NationalDisabilityInsuranceScheme #NDIA #wheelchair
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Designer featured on Gardening Australia stripped of NDIS support funding https://www.allforgardening.com/1846147/designer-featured-on-gardening-australia-stripped-of-ndis-support-funding/ #DuchenneMuscularDystrophy #garden #gardening #GardeningAustralia #JoshuaRuff #NationalDisabilityInsuranceScheme #NDIA #wheelchair
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https://www.europesays.com/ch/87167/ Another setback for Enspryng as Roche halts Duchenne development #ClinicalTrial #DuchenneMuscularDystrophy(DMD) #ENSPRYNG #FiercePharmaHomepage #Pharma #Roche
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https://www.europesays.com/ch/86498/ Roche to launch another Elevidys study, eying EU approval #Cell&GeneTherapy #Duchenne #DuchenneMuscularDystrophy(DMD) #Elevidys #EuropeanMedicinesAgency #FiercePharmaHomepage #GeneTherapy #Pharma #Roche #SareptaTherapeutics
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The structure of spectrin-like repeat 24 of human dystrophin is in excellent agreement with the AlphaFold model, supporting AlphaFold modeling of all 24 dystrophin spectrin-like repeats #Dystrophin #SpectrinLikeRepeat #DuchenneMuscularDystrophy https://doi.org/10.1107/S2053230X26003262
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The structure of spectrin-like repeat 24 of human dystrophin is in excellent agreement with the AlphaFold model, supporting AlphaFold modeling of all 24 dystrophin spectrin-like repeats #Dystrophin #SpectrinLikeRepeat #DuchenneMuscularDystrophy https://doi.org/10.1107/S2053230X26003262
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The structure of spectrin-like repeat 24 of human dystrophin is in excellent agreement with the AlphaFold model, supporting AlphaFold modeling of all 24 dystrophin spectrin-like repeats #Dystrophin #SpectrinLikeRepeat #DuchenneMuscularDystrophy https://doi.org/10.1107/S2053230X26003262
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https://www.europesays.com/ch/83342/ Novartis delpacibart braxlosiran (del-brax) Phase I/II study in facioscapulohumeral muscular dystrophy (FSHD) meets primary biomarker endpoint #ClinicalStudies #DuchenneMuscularDystrophy #FSHD #MuscleDamage #MuscularDystrophy #NeuromuscularDisease #Novartis #patients #PhaseIII #PhaseIIITrial #SpinalMuscularAtrophy
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https://www.europesays.com/ch/81803/ Novartis Reports Positive Data for Neuromuscular Disorder Drug #CreatineKinase #DuchenneMuscularDystrophy #FSHD #GeneTherapy #MuscleDamage #MuscularDystrophy #Novartis #PhaseIIITrial #ZacksInvestmentResearch
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https://www.europesays.com/ch/81262/ Novartis delpacibart braxlosiran (del-brax) Phase I/II study in facioscapulohumeral muscular dystrophy (FSHD) meets primary biomarker endpoint #ClinicalStudies #DuchenneMuscularDystrophy #FSHD #MuscleDamage #MuscularDystrophy #NeuromuscularDisease #Novartis #patients #PhaseIII #PhaseIIITrial #SpinalMuscularAtrophy
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AstraZeneca’s ‘extensive’ obesity drug push; $125M for ultrasound-aided genetic medicine
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free…
#EuropeSays #Britain #Europe #EU #AstraZeneca #BenFidler #bloodsugarlevels #Duchennemusculardystrophy #Obesity
https://www.europesays.com/britain/60215/ -
https://www.europesays.com/britain/60215/ AstraZeneca’s ‘extensive’ obesity drug push; $125M for ultrasound-aided genetic medicine #AstraZeneca #BenFidler #BloodSugarLevels #DuchenneMuscularDystrophy #Obesity
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https://www.europesays.com/ch/62092/ Sarepta swings to Q1 2026 profit on Roche revenue #DuchenneMuscularDystrophy #Elevidys #NonGAAPNetIncome #Q12026Earnings #QuarterlyResults #Roche #RocheCollaboration #SareptaTherapeutics #SRPT
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https://www.europesays.com/ch/51103/ Roche plots route to EMA approval for DMD gene therapy #BioWorld #ChugaiPharmaceuticalCoLtd #DuchenneMuscularDystrophy #Elevidys #EMA #Europe #GeneTherapy #JAPAN #Musculoskeletal #Regulatory #Roche #RocheAG #SareptaTherapeuticsInc
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https://www.europesays.com/ch/50047/ Roche to start new Elevidys study following setback in Europe #DuchenneMuscularDystrophy #DuchennePatients #Europe #GeneTherapy #RegulatoryApproval #Roche #SareptaTherapeutics #StudyResults
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They thought their son lived an isolated life. But when he died, friends showed up in droves.
https://fed.brid.gy/r/https://www.upworthy.com/mats-steen-ibelin-world-of-warcraft-ex1
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They thought their son lived an isolated life. But when he died, friends showed up in droves.
https://web.brid.gy/r/https://www.upworthy.com/mats-steen-ibelin-world-of-warcraft-ex1
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They thought their son lived an isolated life. But when he died, friends showed up in droves.
https://web.brid.gy/r/https://www.upworthy.com/mats-steen-ibelin-world-of-warcraft-ex1
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They thought their son lived an isolated life. But when he died, friends showed up in droves.
https://web.brid.gy/r/https://www.upworthy.com/mats-steen-ibelin-world-of-warcraft-ex1
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https://www.europesays.com/uk/570996/ Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information #AcuteLiverFailure #AmbulatoryPatients #DMDGene #DuchenneMuscularDystrophy #ELEVIDYS #Health #IntravenousInfusion #LiverInjury #Medication #PrescribingInformation #SareptaTherapeutics #UK #UnitedKingdom
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Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information
CAMBRIDGE, Mass., November 14, 2025–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for …
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Medication #acuteliverfailure #ambulatorypatients #DMDgene #duchennemusculardystrophy #ELEVIDYS #Health #intravenousinfusion #Liverinjury #prescribinginformation #SareptaTherapeutics
https://www.newsbeep.com/us/292160/ -
Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information
CAMBRIDGE, Mass., November 14, 2025–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for …
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Medication #acuteliverfailure #ambulatorypatients #DMDgene #duchennemusculardystrophy #ELEVIDYS #Health #intravenousinfusion #Liverinjury #prescribinginformation #SareptaTherapeutics
https://www.newsbeep.com/us/292160/ -
Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information
CAMBRIDGE, Mass., November 14, 2025–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases,…
#NewsBeep #News #Medication #acuteliverfailure #ambulatorypatients #DMDgene #duchennemusculardystrophy #ELEVIDYS #Health #intravenousinfusion #Liverinjury #prescribinginformation #sareptatherapeutics #UK #UnitedKingdom
https://www.newsbeep.com/uk/263365/ -
https://www.europesays.com/ie/181196/ Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information #AcuteLiverFailure #AmbulatoryPatients #DMDGene #DuchenneMuscularDystrophy #Éire #ELEVIDYS #Health #IE #IntravenousInfusion #Ireland #LiverInjury #Medication #PrescribingInformation #SareptaTherapeutics
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Satellos to Present DMD Drug SAT-3247 Phase 1b Data at WMS 2025
09/29/2025…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #BreakingNews #biotechnology #clinicaldata #DMD #duchennemusculardystrophy #FrankGleeson #Headlines #MSCLF #musclerepair #Phase1bclinicaltrial #SAT-3247 #Satellos #Topstories #TopStories #WildonFarwell #WMS2025 #WorldMuscleSocietyCongress
https://www.newsbeep.com/us/189681/ -
Satellos to Present DMD Drug SAT-3247 Phase 1b Data at WMS 2025
09/29/2025…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #BreakingNews #biotechnology #clinicaldata #DMD #duchennemusculardystrophy #FrankGleeson #Headlines #MSCLF #musclerepair #Phase1bclinicaltrial #SAT-3247 #Satellos #Topstories #TopStories #WildonFarwell #WMS2025 #WorldMuscleSocietyCongress
https://www.newsbeep.com/us/189681/ -
Duchenne Muscular Dystrophy Market to Witness Significant
New York, USA, July 28, 2025 (GLOBE NEWSWIRE) — Duchenne Muscular Dystrophy Market to Witness Significant Expansion During…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #DelveInsightBusinessResearchLLP #DMDCompanies #DMDDrugs #DMDMarket #DMDTherapies #DMDTrials #duchennemusculardystrophy #Science
https://www.newsbeep.com/us/43213/ -
Duchenne Muscular Dystrophy Market to Witness Significant
New York, USA, July 28, 2025 (GLOBE NEWSWIRE) — Duchenne Muscular Dystrophy Market to Witness Significant Expansion During…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #DelveInsightBusinessResearchLLP #DMDCompanies #DMDDrugs #DMDMarket #DMDTherapies #DMDTrials #duchennemusculardystrophy #Science
https://www.newsbeep.com/us/43213/ -
New DMD Therapy Inhibits Glutamate Dehydrogenase 1
Credit: Sinhyu / iStock / Getty Images Plus Researchers at the VIB-KU Leuven Center for Brain and Disease…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #duchennemusculardystrophy #enzymes #muscles #news&features #Patientcare #pharmacology #Science #topics #translationalresearch
https://www.newsbeep.com/us/21194/ -
New DMD Therapy Inhibits Glutamate Dehydrogenase 1
Credit: Sinhyu / iStock / Getty Images Plus Researchers at the VIB-KU Leuven Center for Brain and Disease…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #duchennemusculardystrophy #enzymes #muscles #news&features #Patientcare #pharmacology #Science #topics #translationalresearch
https://www.newsbeep.com/us/21194/ -
New DMD Therapy Inhibits Glutamate Dehydrogenase 1
Credit: Sinhyu / iStock / Getty Images Plus Researchers at the VIB-KU Leuven Center for Brain and Disease…
#NewsBeep #News #US #USA #UnitedStates #UnitedStatesOfAmerica #Genetics #duchennemusculardystrophy #enzymes #muscles #news&features #Patientcare #pharmacology #Science #topics #translationalresearch
https://www.newsbeep.com/us/21194/ -
Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - https://arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda
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Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - https://arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda
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Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - https://arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda
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Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - https://arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda
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Top FDA official overrules staff to approve gene therapy that failed trial - Enlarge / Dr. Peter Marks, Director of the Center for Biologics Evaluat... - https://arstechnica.com/?p=2032919 #duchennemusculardystrophy #clinicaltrials #clinicaldata #fdaapproval #genetherapy #petermarks #elevidys #science #sarepta #fda
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Thrilled to share our story on the first rationally designed #covalent #HDAC inhibitor! YSR734 uses an SNAr-reactive electrophile to engage a noncatalytic nitrosylation site cysteine in #HDAC2, showing in vitro activity in AML & #DuchenneMuscularDystrophy!
https://pubs.acs.org/doi/10.1021/acs.jmedchem.3c01236 -
Thrilled to share our story on the first rationally designed #covalent #HDAC inhibitor! YSR734 uses an SNAr-reactive electrophile to engage a noncatalytic nitrosylation site cysteine in #HDAC2, showing in vitro activity in AML & #DuchenneMuscularDystrophy!
https://pubs.acs.org/doi/10.1021/acs.jmedchem.3c01236 -
Thrilled to share our story on the first rationally designed #covalent #HDAC inhibitor! YSR734 uses an SNAr-reactive electrophile to engage a noncatalytic nitrosylation site cysteine in #HDAC2, showing in vitro activity in AML & #DuchenneMuscularDystrophy!
https://pubs.acs.org/doi/10.1021/acs.jmedchem.3c01236 -
Data Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical #data and #records indicate matched #genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
https://sciencebusiness.technewslit.com/?p=44426
#News #Science #Business #ClinicalTrials #DuchenneMuscularDystrophy #Neuroscience #Neurology #ControlGroup #Placebo #Statistics #RareDisease #InheritedDisease #RealWorldData
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Data Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical #data and #records indicate matched #genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
https://sciencebusiness.technewslit.com/?p=44426
#News #Science #Business #ClinicalTrials #DuchenneMuscularDystrophy #Neuroscience #Neurology #ControlGroup #Placebo #Statistics #RareDisease #InheritedDisease #RealWorldData
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Data Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical #data and #records indicate matched #genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
https://sciencebusiness.technewslit.com/?p=44426
#News #Science #Business #ClinicalTrials #DuchenneMuscularDystrophy #Neuroscience #Neurology #ControlGroup #Placebo #Statistics #RareDisease #InheritedDisease #RealWorldData
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Data Support Placebo Group Alternatives in Rare Disease Trials
Findings from a review of medical #data and #records indicate matched #genetic control groups may not be needed in clinical trials testing treatments for Duchenne muscular dystrophy.
https://sciencebusiness.technewslit.com/?p=44426
#News #Science #Business #ClinicalTrials #DuchenneMuscularDystrophy #Neuroscience #Neurology #ControlGroup #Placebo #Statistics #RareDisease #InheritedDisease #RealWorldData
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Huge thanks @ElectricWill for your wonderful donation 🙌🏻 to our fundraising campaign for #DuchenneMuscularDystrophy
I’m really looking forward to connecting with you more on here!
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Huge thanks @ElectricWill for your wonderful donation 🙌🏻 to our fundraising campaign for #DuchenneMuscularDystrophy
I’m really looking forward to connecting with you more on here!
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Huge thanks @ElectricWill for your wonderful donation 🙌🏻 to our fundraising campaign for #DuchenneMuscularDystrophy
I’m really looking forward to connecting with you more on here!