Action Duchenne

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Work with partner organisations to run clinical trials of approved drugs that show promise for the treatment of Duchenne Muscular Dystrophy. Parents of boys spend a lot of money on supplements that are believed to provide benefit. Pilot Trials will provide the qualitative results they deserve.

history Campaign has now closed

It ran from 10:00 AM, 6 December 2010 to 10:00 AM, 4 January 2011

open_in_new https://www.actionduchenne.org/

Registered Charity in England and Wales (1101971)

Check mark Match funded

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    Category

  • Medical ResearchMedical Research

    Helping

Location

Situation

Currently the only medication available is steroids which may (at best) keep diagnosed children walking for additional two years and are accompanied by negative side effects including radio weight gain and loss of bone density. Bottom line: the disease remains 100% fatal, with most boys dying in the teens or 20s. There is not one approved drug offered to those suffering with Duchenne.

Solution

We will run clinical trials on FDA approved drug IGF1 which shows promise in the treatment of Duchenne. By supporting this trial we are finally giving boys with Duchenne a chance to save their muscles by triggering continual regeneration of them and building muscle strength. This will help to slow the severe and fast muscle wasting which is the primary cause of early death.