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The view from the trenches – Advocating for your rare disease child
This is Rebecca’s story – she presented this to a conference recently. Rebecca lives in Glasgow, Scotland, United Kingdom....
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If I meet the Exclusion Criteria, is that a bad thing?
In my post a few days ago, I mentioned that to take part in a clinical trial, you will...
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Are You Happy? Life According to Sam Berns
Sampson Gordon “Sam” Berns was born on October 23, 1996 and died on January 10, 2014. He was less...
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Will I meet the inclusion criteria?
Of the 7000 rare diseases, most do not have treatments or effective treatments. Most patients with rare diseases will...
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Find clinical trials for your rare disease / your child’s rare disease
There are 7000 rare diseases, most of which lack treatments, or effective treatments. The purpose of todays Blog post...
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The Right to Try – Let’s Talk About the Good
The Right to Try law was signed by President Trump on 30 May 2018. Whilst there are bad and a just plain...
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The Right to Try – The Good, the Bad, and the Just Plain Ugly
The Right to Try law was signed into law by President Trump on 30 May 2018. It is appropriate...
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Autism Spectrum Disorder – The Future
Autism Spectrum Disorder is one of the most challenging rare disease disorders to develop new treatments for. Nevertheless, it is good...
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The Charlie Gard Case – Lessons to be Learnt
Background For those of you that have not been following the case of Charlie Gard, Charlie was a baby, who...
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The Value of a Second Opinion
… cannot be underestimated. In the case of Charlie Gard it could mean the difference between having his life...