Duchenne muscular dystrophy

News

Nippon Shinyaku’s DMD drug fails confirmatory trial

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

News

EU rules PTC’s Duchenne drug can stay on the market

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

News

Pfizer pauses DMD gene therapy trial after boy’s death

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

News

FDA okays Italfarmaco’s non-steroidal DMD therapy Duvyzat

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

News

Santhera debuts DMD drug Agamree in Germany

Santhera’s new treatment for Duchenne muscular dystrophy Agamree has been launched in its first market, Germany, following its EU approval last month.

News

Despite trial miss, Sarepta plans new filing for DMD therapy

Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy has failed to meet its primary objective in a phase 3 trial, but the company says it will file to extend

Nippon Shinyaku’s DMD drug fails confirmatory trial

EU rules PTC’s Duchenne drug can stay on the market

Pfizer pauses DMD gene therapy trial after boy’s death

FDA okays Italfarmaco’s non-steroidal DMD therapy Duvyzat

Santhera debuts DMD drug Agamree in Germany

Despite trial miss, Sarepta plans new filing for DMD therapy

Pharma in a spin as it tries to gauge Trump tariffs impact

Health Trends 2025: Reimagining What's Possible

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Duchenne muscular dystrophy

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