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September 29, 2021

Pfizer adds to gene therapy woes

But another safety scare for the big pharma’s Duchenne muscular dystrophy project could be good news for Sarepta.

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June 07, 2019

Vertex’s second transformation: Duchenne time

The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.

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September 06, 2017

Sarepta hopes to pull off the same trick twice

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October 14, 2016

Snippet roundup: Biomarin boosted, Synairgen stung

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October 06, 2016

Therapy focus – Duchenne assets soar in the wake of Exondys 51 approval

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February 24, 2016

The US FDA shows its teeth

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December 11, 2013

ASH – Interesting theories, but doubts on novel myelofibrosis projects remain

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