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 from Maze Therapeutics.

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

Motivated patients with rare diseases are using social media widely, making it an effective way to reach patients for trial ...