Duchenne muscular dystrophy | pharmaphorum

Wednesday 8 January 2025

Channels

Content type

Categories

Categories

Sort by

Order

Duchenne muscular dystrophy

News

DNA sequence

Summit shares plunge nearly 80% after DMD drug fails

Summit Therapeutics has axed development of its Duchenne muscular dystrophy drug ezutromid after a spectacular failure in phase 2, causing the UK biotech's shares to plunge.

Views & Analysis

At the heart of patient centric care

Improving study recruitment with patient-centric trial endpo...

Everyone wants to find a cure for a debilitating long-term condition, but what can often be overlooked is the value in being able to first make life more manageable, comfortable or enjoyabl

News

Sarepta goes up, PTC goes down – DMD research sways the mark...

US biotech Sarepta's shares jumped by as much as 60% after a gene therapy produced dramatic results in three boys with the muscle wasting disease Duchenne muscular dystrophy (DMD).

Views & Analysis

Group of people holding hand together in the park

Living with DMD: Ensuring equal access to quality of life

Making sure everyone with DMD gets access to treatments and the ‘essential’ interventions that can make life worth living is key.

Views & Analysis

Film-movie-clapperboard

Lights, camera, action – DMD and chasing your dreams

People with a DMD diagnosis must believe this isn’t a sign to roll the end credits on their lives, says Luca Buccella.

Views & Analysis

Jon-Hastie-DMD

Living with DMD: Inspiring each other’s futures

Diagnosed with Duchenne muscular dystrophy (DMD) at three years old, Dr Jon Hastie grew up n