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Home
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» DMD
DMD
Regenxbio data suggest ‘niche’ in Duchenne gene therapy
BioPharma Dive
Thu, 03/7/24 - 11:05 am
Regenxbio
Duchenne Muscular Dystrophy
DMD
RGX-202
Sarepta Therapeutics
Elevidys
Sarepta's next DMD drug seems to get the job done, but Elevidys casts long shadow
Fierce Biotech
Mon, 01/29/24 - 10:25 pm
Sarepta Therapeutics
DMD
SRP-5051
Elevidys
PTC's re-examination bid for DMD drug Translarna falls short in Europe
Fierce Pharma
Fri, 01/26/24 - 11:10 am
PTC Therapeutics
EMA
Europe
Translarna
DMD
Santhera debuts DMD drug Agamree in Germany
Pharmaphorum
Mon, 01/15/24 - 09:37 am
Santhera
Duchenne Muscular Dystrophy
DMD
Agamree
Germany
Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Fierce Pharma
Thu, 11/2/23 - 05:15 pm
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
Elevidys
gene therapy
Sarepta’s disappointing data put the FDA in a difficult spot
Stat
Tue, 10/31/23 - 10:16 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
FDA
gene therapy
FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
BioSpace
Fri, 10/27/23 - 11:47 am
Santhera Pharmaceuticals
Catalyst Pharmaceuticals
Agamree
FDA
DMD
Duchenne Muscular Dystrophy
Santhera closes on first approval for DMD drug vamorolone
Pharmaphorum
Fri, 10/13/23 - 10:23 am
Santhera
Duchenne Muscular Dystrophy
DMD
vamorolone
EMA
PTC deepens layoffs as DMD drug faces loss of authorization across Europe
Fierce Biotech
Fri, 09/29/23 - 10:19 am
PTC Therapeutics
DMD
layoffs
Europe
Translarna
After FDA setback, PTC Therapeutics' DMD drug Translarna faces possible market withdrawal in Europe
Fierce Pharma
Fri, 09/15/23 - 11:49 am
PTC Therapeutics
DMD
Translarna
Europe
10 clinical trials to watch in the second half of 2023
BioPharma Dive
Mon, 08/7/23 - 10:32 am
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
5 Highly Anticipated FDA Decisions for Rare Diseases Coming This Year
BioSpace
Fri, 07/28/23 - 11:46 am
Ipsen
palovarotene
fibrodysplasia ossificans progressiva
Regeneron
CHAPLE disease
pozelimab
Biomarin
Voxzogo
achondroplasia
Santhera Pharmaceuticals
vamorolone
DMD
Springworks
nirogacestat
desmoid tumors
Sarepta prices Duchenne gene therapy at $3.2M
BioPharma Dive
Fri, 06/23/23 - 09:44 am
Sarepta Therapeutics
drug pricing
DMD
Duchenne Muscular Dystrophy
Elevidys
FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
BioSpace
Thu, 06/22/23 - 10:51 pm
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
FDA
Elevidys
gene therapy
FDA delays decision on Duchenne gene therapy and considers narrower approval
BioPharma Dive
Wed, 05/24/23 - 11:21 am
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
Sarepta Enters Uncharted Territory with Adcomm Approval
BioSpace
Mon, 05/22/23 - 10:22 am
Sarepta Therapeutics
SRP-9001
DMD
Duchenne Muscular Dystrophy
FDA
gene therapy
DMD gene therapy death exposes risks of treating older patients
Fierce Biotech
Mon, 05/22/23 - 10:09 am
Cure Rare Disease
gene therapy
clinical trials
DMD
patient deaths
FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
Fierce Biotech
Mon, 05/15/23 - 11:18 am
Sarepta Therapeutics
FDA
DMD
gene therapy
Duchenne Muscular Dystrophy
FDA reviewers flag concerns over Sarepta's gene therapy for DMD
Yahoo/Reuters
Wed, 05/10/23 - 10:08 am
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
Fierce Biotech
Wed, 05/3/23 - 11:39 am
Sarepta Therapeutics
FDA
SRP-9001
DMD
Duchenne Muscular Dystrophy
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