DMD

Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Fierce Pharma
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
Elevidys
gene therapy
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Sarepta’s disappointing data put the FDA in a difficult spot
Sarepta’s disappointing data put the FDA in a difficult spot
Stat
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
FDA
gene therapy
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FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
BioSpace
Santhera Pharmaceuticals
Catalyst Pharmaceuticals
Agamree
FDA
DMD
Duchenne Muscular Dystrophy
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Santhera closes on first approval for DMD drug vamorolone
Santhera closes on first approval for DMD drug vamorolone
Pharmaphorum
Santhera
Duchenne Muscular Dystrophy
DMD
vamorolone
EMA
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PTC deepens layoffs as DMD drug faces loss of authorization across Europe
PTC deepens layoffs as DMD drug faces loss of authorization across Europe
Fierce Biotech
PTC Therapeutics
DMD
layoffs
Europe
Translarna
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After FDA setback, PTC Therapeutics' DMD drug Translarna faces possible market withdrawal in Europe
After FDA setback, PTC Therapeutics' DMD drug Translarna faces possible market withdrawal in Europe
Fierce Pharma
PTC Therapeutics
DMD
Translarna
Europe
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10 clinical trials to watch in the second half of 2023
10 clinical trials to watch in the second half of 2023
BioPharma Dive
clinical trials
Sarepta Therapeutics
DMD
Elevidys
Bridge Bio
ATTR-CM
acoramidis
Sanofi
Merck KGaA
MS
tolebrutinib
Pfizer
obesity
danuglipron
Novo Nordisk
Ozempic
Verve Therapeutics
heterozygous familial hypercholesterolemia
Roche
lung cancer
tiragolumab
Vertex Pharmaceutical
VS-548
pain
JNJ
Bristol Myers Squibb
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5 Highly Anticipated FDA Decisions for Rare Diseases Coming This Year
5 Highly Anticipated FDA Decisions for Rare Diseases Coming This Year
BioSpace
Ipsen
palovarotene
fibrodysplasia ossificans progressiva
Regeneron
CHAPLE disease
pozelimab
Biomarin
Voxzogo
achondroplasia
Santhera Pharmaceuticals
vamorolone
DMD
Springworks
nirogacestat
desmoid tumors
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Sarepta prices Duchenne gene therapy at $3.2M
Sarepta prices Duchenne gene therapy at $3.2M
BioPharma Dive
Sarepta Therapeutics
drug pricing
DMD
Duchenne Muscular Dystrophy
Elevidys
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FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
BioSpace
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
FDA
Elevidys
gene therapy
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FDA delays decision on Duchenne gene therapy and considers narrower approval
FDA delays decision on Duchenne gene therapy and considers narrower approval
BioPharma Dive
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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Sarepta Enters Uncharted Territory with Adcomm Approval
Sarepta Enters Uncharted Territory with Adcomm Approval
BioSpace
Sarepta Therapeutics
SRP-9001
DMD
Duchenne Muscular Dystrophy
FDA
gene therapy
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DMD gene therapy death exposes risks of treating older patients
DMD gene therapy death exposes risks of treating older patients
Fierce Biotech
Cure Rare Disease
gene therapy
clinical trials
DMD
patient deaths
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FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
Fierce Biotech
Sarepta Therapeutics
FDA
DMD
gene therapy
Duchenne Muscular Dystrophy
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FDA reviewers flag concerns over Sarepta's gene therapy for DMD
FDA reviewers flag concerns over Sarepta's gene therapy for DMD
Yahoo/Reuters
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
Fierce Biotech
Sarepta Therapeutics
FDA
SRP-9001
DMD
Duchenne Muscular Dystrophy
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Go or no go? Sarepta’s gene therapy faces scrutiny
Go or no go? Sarepta’s gene therapy faces scrutiny
EP Vantage
Sarepta Therapeutics
gene therapy
SRP-9001
Roche
DMD
Duchenne Muscular Dystrophy
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Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Yahoo/Reuters
Sarepta Therapeutics
FDA
gene therapy
DMD
Duchenne Muscular Dystrophy
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FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy
FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy
BioSpace
FDA
Sarepta Therapeutics
DMD
gene therapy
Duchenne Muscular Dystrophy
SRP-9001
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Duchenne muscular dystrophy: major trials and events to watch in 2023
Duchenne muscular dystrophy: major trials and events to watch in 2023
Clinical Trials Arena
DMD
Duchenne Muscular Dystrophy
clinical trials
gene therapy
FDA
Sarepta Therapeutics
SRP-9001
FibroGen
pamrevlumab
Capricor
CAP-1002
Santhera
ReveraGen
vamorolone
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