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achondroplasia

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FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A

Approximately 2,500 patients are expected to be eligible to receive the one-time therapy

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BioMarin’s application for haemophilia A gene therapy accepted by FDA

If approved, the treatment would be the first gene therapy in the US for this indication

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EC approves BioMarin’s gene therapy Roctavian for severe haemophilia A

Approximately 20,000 adults are affected by haemophilia A across more than 70 countries in Europe, the Middle East and Africa

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FDA keeps BioMarin’s gene therapy for phenylketonuria on hold

The decision follows data suggesting BioMarin’s experimental gene therapies may have the potential to progress cancers

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FDA pauses BioMarin gene therapy trial

The study for BMN 307 in genetic disease phenylketonuria will be paused while malignancies in mice are explored

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Presidential hopeful Bernie Sanders criticises BioMarin’s gene therapy price

Vowed to break patent if company goes ahead with proposed price

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Sangamo steps up BioMarin chase with haemophilia gene therapy data

Updated results presented at ASH 2019

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NICE approves BioMarin’s Brineura after initial setback

First treatment for rare disease

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BioMarin ticks over in Q2 ahead of gene therapy filing

Haemophilia candidate valrox will be a game-changer for company

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ACPs to lead gene therapies in haemophilia market growth

New therapies to hit existing players

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BioMarin eyes haemophilia therapy filing, but data scares investors

Doubts remain on longer term efficacy

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EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

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