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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

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

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BioMarin opens €38m expansion at Irish manufacturing site

BioMarin opens €38m expansion at Irish manufacturing site

Biopharma Reporter

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

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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

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JPM Day 1: Highlights

JPM Day 1: Highlights

BioSpace

Sarepta Therapeutics

Arrowhead Pharmaceuticals

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

Alzheimer's disease

Acadia Pharmaceuticals

Gilead Sciences

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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

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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

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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

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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

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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

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

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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

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

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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

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

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

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