Promising news for CMT1As

Published on March 27th, 2020

Pharma company Pharnext, raises 7.7 M euros to support the clinical development of PXT3003 treatment for Charcot Marie Tooth disease. The funding will help finalize the study protocol for an upcoming Phase 3 trial.

The company announces: “Extension study dosing will continue until PXT3003 is commercially available. Detailed study results are expected later this year”.

We also remember that recently, PXT3003 has been granted Promising Innovative Medicine (PIM) by the UK health authorities and that, in 2014, the EMA and FDA granted orphan drug designation to PXT3003 for the treatment of CMT1A in adults.

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