Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD) will be an option for many more patients in the US after the FDA expanded the breadth of its approved labelling.
The future of Nippon Shinyaku’s Duchenne muscular dystrophy (DMD) therapy Viltepso has been thrown into doubt after it failed a clinical trial designed to upgrade its 2020
Going against the advice of its own advisors, the European Commission has said that PTC Therapeutics’ Translarna therapy for Duchenne muscular dystrophy (DMD) should stay
The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to paus
Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD), becoming the first non-steroidal option for
In a new white paper from the World Without Disease initiative, a 2024 update is provided of the current endeavours and developments that have occurred since inaugural discussions last year