We're all Joining Jacks fight against Duchenne Musculare Dystrophy, are you?

Guest Blog by Ruth Le Gal

Leo was 5 when I realized that there was something wrong with his legs.  As a physiotherapist I knew a little bit about Duchenne Muscular Dystrophy, and it only took me a few minutes of researching on the internet to work out that he was almost certainly going to be diagnosed with this horrific and devastating illness.  DNA testing soon confirmed my worst fears.  No words can really explain the devastation I have felt over the last 3 years as I have watched my son waste away.  If you have a child with Duchenne then you know how it feels.  If you don’t, then there just isn’t any way to fully explain to you the complete and utter misery of living with this terrible disease.

Of course I tried every possible thing to help my son.  I gave him vitamin C, vitamin D, calcium, magnesium, omega 3, green tea, Q10, idebenone and steroids.  I attended a million hospital appointments.  In desperation, I tried Vecttor therapy, healing therapy and hydrotherapy.  I used my physiotherapy skills to massage and stretch his little legs.  Nothing helped.  He simply grew weaker and weaker every single day until he was just a pale and fragile version of the boy that he had once been.

Then one day a miracle happened.  Leo was allowed to start taking a medication called Translarna.  Within a few weeks it became clear that he was no longer deteriorating.  The relentless and devastating decline that I had watched for 3 long and terrible years had finally stopped, his condition had stabilized and he was no longer getting worse.  Although he is still weak and there are many things he can’t do, my precious son is no longer wasting away.  Thanks to Translarna.

Every child that needs this medication should be getting it now.  Translarna is not a cure for Duchenne, but it is a very effective treatment that halts or dramatically slows down the deterioration caused by the disease.  In the same way, although it is not a cure, Insulin is a very effective treatment for Diabetes.  It would be unthinkable to deny children with Diabetes access to their insulin, and it is brutal and cruel to deny children with Duchenne access to their Translarna.

This Christmas let’s make a miracle happen.  Let’s make Translarna available to all the children in the UK who need it.  No more wasting away, no more Duchenne children waiting for the medications that they so desperately need.  Ask your MP to take this up with the Secretary of State for Health, and add your name to the petition asking the NHS to make this medication available for all the children who need it.

http://you.38degrees.org.uk/petitions/fund-first-treatment-for-duchenne-muscular-dystrophy

 Leo canoeing

 

 

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