Rare Disease

R&D

A closer look at gene therapy with PTC Therapeutics

In the latest instalment of the pharmaphorum podcast, editor-in-chief Jonah Comstock connects with Kylie O’Keefe to talk about gene therapy in rare diseases.

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PTC eyes filings for PKU therapy after phase 3 win

PTC Therapeutics is on track to file for approval of a new therapy for rare disease phenylketonuria after a positive phase 3 readout that could extend its product portfoli

R&D

The role of a Research Consulting Organisation in educating ...

How can RCOs help biotech, pharma, and device innovators bring life-saving drugs to market sooner and more cost effectively?

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Sanofi finds new Pompe disease therapy in Maze

Sanofi is already a big player in the Pompe disease market with injectable drugs like Nexviazyme and Myozyme/Lumizyme, but has now set its sights on an oral drug candidate

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