Great news for the Duchenne Community

October 14, 2023 11:02 am

EMA recommends approval of first ever treatment for all DMD patients

Today The European Medicines Agency (EMA) has recommended the approval of Vamorolone, the first ever treatment for all patients with Duchenne muscular dystrophy (DMD).

Vamorolone is an alternative to corticosteroids, which are recommended for use in the treatment of DMD as they can help to reduce muscle inflammation and maintain muscle strength for boys.

However Corticosteroids have several negative side effects, including weight gain and ostioporosis. Vamorolone will help to reduce muscle inflammation and strength with fewer side-effects.

Alex’s Wish invested in the early stages of the development of Vamorolone

We, alongside other members of the Duchenne Community invested in this project in pre-clinical trials, developing an alternative to steroids that will be chemically altered to change a series of properties that may show optimization for Duchenne muscular dystrophy and other chronic inflammatory disorders.

The success of this project quickly moved on to clinical trials that was supported by Duchenne UK to get to the stage we are at today.

Although this decision does not mean that Vamorolone is available in the UK it is currently under review, NICE will review Vamorolone to decide if it is clinically effective and cost effective for NHS patients. A decision is expected in mid 2024. Duchenne UK will be part of the NICE appraisal process, representing the Duchenne Community. We will keep you up to date with any progress.

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