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Biomarin
PTC, chasing BioMarin, posts phase 3 rare disease win but fails to silence skeptics
PTC, chasing BioMarin, posts phase 3 rare disease win but fails to silence skeptics
Fierce Biotech
PTC Therapeutics
Biomarin
PKU
clinical trials
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Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch
Gene Therapies Are Still Hampered By Substantial Delays Between Approval And Launch
Forbes
gene therapy
drug approvals
Biomarin
Bluebird Bio
FDA
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BioMarin opens €38m expansion at Irish manufacturing site
BioMarin opens €38m expansion at Irish manufacturing site
Biopharma Reporter
Biomarin
Ireland
drug manufacturing
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BioMarin Answers BridgeBio's Challenge with sNDA for Achondroplasia
BioMarin Answers BridgeBio's Challenge with sNDA for Achondroplasia
BioSpace
Biomarin
BridgeBio
achondroplasia
FDA
Voxzogo
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FDA delays review of BioMarin's hemophilia gene therapy after data update
FDA delays review of BioMarin's hemophilia gene therapy after data update
Endpoints
Biomarin
gene therapy
hemophilia A
FDA
valrox
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Go or no go? Vaccines and neurology up for discussion
Go or no go? Vaccines and neurology up for discussion
EP Vantage
FDA
Acadia Pharmaceuticals
Apellis Pharmaceuticals
AstraZeneca
Biogen
Biomarin
Cidara Therapeutics
Emergent BioSolutions
GSK
Incyte
Ionis Pharmaceuticals
Melinta Therapeutics
Mundipharma
Novartis
Pfizer
Pharming
Regeneron
Roche
vaccines
RSV
ALS
COVID-19
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JPM Day 1: Highlights
JPM Day 1: Highlights
BioSpace
JPMHC 2023
Sarepta Therapeutics
Biomarin
Alnylam
JNJ
Arrowhead Pharmaceuticals
Novartis
Bristol Myers Squibb
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5 FDA decisions to watch in the first quarter
5 FDA decisions to watch in the first quarter
BioPharma Dive
FDA
Eli Lilly
donanemab
Alzheimer's disease
Menareni
Elacestrant
breast cancer
Biomarin
Roctavian
hemophilia A
Acadia Pharmaceuticals
trofinetide
Rett Syndrome
Gilead Sciences
Trodelvy
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Biotech’s key upcoming clinical results
Biotech’s key upcoming clinical results
EP Vantage
clinical trials
Acadia Pharmaceuticals
Argenx
Arrowhead Pharmaceuticals
Biomarin
BioNTech
Daiichi Sankyo
Day One
EQRx
Fate Therapeutics
FibroGen
Gilead Sciences
Idorsia
Immunogen
Intellia Therapeutics
Karuna Therapeutics
Moderna Therapeutics
Regeneron
Roche
Travere Therapeutics
Vaxcyte
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BioMarin's hemophilia gene therapy faces further delays over fresh FDA requests
BioMarin's hemophilia gene therapy faces further delays over fresh FDA requests
Fierce Biotech
Biomarin
gene therapy
FDA
hemophilia
Roctavian
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Cost watchdog ICER backs multimillion-dollar price tags for BioMarin, CSL hemophilia gene therapies
Cost watchdog ICER backs multimillion-dollar price tags for BioMarin, CSL hemophilia gene therapies
Fierce Pharma
ICER
drug pricing
gene therapy
Biomarin
CSL
Bluebird Bio
hemophilia
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BioMarin gets FDA decision date for hemophilia gene therapy, but questions on review remain
BioMarin gets FDA decision date for hemophilia gene therapy, but questions on review remain
BioPharma Dive
Biomarin
FDA
hemophilia A
gene therapy
Roctavian
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BioMarin lays off 120 employees to save $50M a year, with US operations hardest hit
BioMarin lays off 120 employees to save $50M a year, with US operations hardest hit
Fierce Biotech
Biomarin
layoffs
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BioMarin Banks on More Robust Data in Hemophilia A Resubmission
BioMarin Banks on More Robust Data in Hemophilia A Resubmission
BioSpace
Biomarin
FDA
hemophilia A
valoctocogene roxaparvovec
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ICER Deems Hemophilia Gene Therapies Worth the $2.5M Price Tag
ICER Deems Hemophilia Gene Therapies Worth the $2.5M Price Tag
BioSpace
hemophilia
gene therapy
ICER
Biomarin
Chugai
Roche
CSL Behring
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BioMarin says PhIII gene therapy study participant's cancer is unrelated to therapy
BioMarin says PhIII gene therapy study participant's cancer is unrelated to therapy
Endpoints
Biomarin
gene therapy
hemophilia A
clinical trials
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BioMarin nets conditional approval for valrox in Europe, marking the first hemophilia gene therapy
BioMarin nets conditional approval for valrox in Europe, marking the first hemophilia gene therapy
Endpoints
Biomarin
hemophilia A
valrox
Europe
gene therapy
Roctavian
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BioMarin's hemophilia gene therapy Roctavian wins conditional EU backing amid FDA delay
BioMarin's hemophilia gene therapy Roctavian wins conditional EU backing amid FDA delay
Fierce Pharma
Biomarin
Roctavian
EMA
FDA
hemophilia A
valoctocogene roxaparvovec
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BioMarin Delays Hemophilia A Therapy Filing as FDA Calls for More Data
BioMarin Delays Hemophilia A Therapy Filing as FDA Calls for More Data
BioSpace
Biomarin
hemophilia A
FDA
valoctocogene roxaparvovec
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BioMarin's Record Q1 Driven by Recently Approved Achondroplasia Therapy
BioMarin's Record Q1 Driven by Recently Approved Achondroplasia Therapy
BioSpace
Biomarin
earnings
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