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....0
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...
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...
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...
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...
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...
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...
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...
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...
The Value of a Second Opinion
… cannot be underestimated. In the case of Charlie Gard it could mean the difference between having his life...