#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/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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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://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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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 rare diseases,…
#NewsBeep #News #Medication #acuteliverfailure #ambulatorypatients #CA #Canada #DMDgene #duchennemusculardystrophy #ELEVIDYS #Health #intravenousinfusion #Liverinjury #prescribinginformation #sareptatherapeutics
https://www.newsbeep.com/ca/283661/ -
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/ -
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/ -
‘Cruel postcode lottery’ facing kids at two Birmingham hospitals just four miles apart
Children with a life-limiting condition face a “cruel postcode lottery” over a new drug – despite being treated…
#Birmingham #UnitedKingdom #UK #GB #England #Headlines #News #Europe #EU #Britain #DMDpatients #DuchenneMuscularDystrophy #FoundationTrust #GreatBritain #HarryReid #MuscularDystrophy #Postcodelottery #RosemaryReid #UniversityHospitals #UniversityofBirmingham
https://www.europesays.com/uk/223109/ -
https://www.europesays.com/uk/223109/ ‘Cruel postcode lottery’ facing kids at two Birmingham hospitals just four miles apart #Birmingham #Britain #DMDPatients #DuchenneMuscularDystrophy #England #FoundationTrust #GreatBritain #HarryReid #MuscularDystrophy #PostcodeLottery #RosemaryReid #UK #UnitedKingdom #UniversityHospitals #UniversityOfBirmingham
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Sarepta Viral Vector Gets FDA Platform Technology Designation
Sarepta Therapeutics Inc. (NASDAQ:SRPT) is one of the best mid cap growth stocks to invest in now. Earlier…
#UnitedStates #US #USA #america #duchennemusculardystrophy #genetherapy #midcapgrowthstocks #musculardystrophy #SareptaTherapeutics #science #technology #unitedstatesofamerica #USFDA #viralvector
https://www.europesays.com/2190686/ -
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 -
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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Our family has a young friend with #DuchenneMuscularDystrophy. We fundraise to help find a cure, - currently believed to be imminent enough to save many thousands of lives.
Each of my children has taken on a challenge to help. Last year, my 11 year-old walked the 102 mile #CotswoldWay. Thanks to people’s incredible generosity, he raised £10k for #DuchenneUK!
I am so proud of him ❤️
I would be so grateful if you could boost this to help us raise awareness. Thank you!
*More info in ALT text*