This is particularly relevant to the rare disease patient whose daily reality is governed by a poorly understood medical condition, where treatments are targeted at symptom suppression, with little known understanding to cause and cure.
And, of course, to have actually attained a diagnosis, is an accomplishment in itself, since on average this takes 4 years.
I wrote about the importance of an early diagnosis, here
I was given a 15month prognosis by my diagnosing doctor, as, there is no cure, and still, there remains no cure.
I am now 13 years free from this regime, with just taking a daily tablet to open my blood vessels and remain indoors at much as possible, to prevent Raynauds attacks.
This required me to have to travel to the Scleroderma Unit in London.
I was able to remain at home in Southport for the trial duration, with the lovely Yvonne, the clinical trial nurse, staying in regular phone contact with me, for that time.
Having a rare disease certainly takes you down life paths which you would never have dreamed of going, as well as, makes you find inner strength, which you never knew you had.
I have always hated needles, and still do, I can not even watch the phlebotomist put the needle into my arm at my current bi-monthly blood tests.
I was required to inject myself with a liquid which was stored in a small vial, which I had just removed from the freezer.
I had several minutes to warm the vial, so that the frozen ingredient became a liquid, draw the liquid into the needle and then inject myself, twice a week, including Christmas Day 2008.
I used my stomach as my injection site, giving myself the reward of cake, to keep my injection site maintained properly.
I would also play my favourite music at that time, to get me in to ‘the zone’ for doing what I needed to get on and do.
Sadly, I understand that the results were not promising enough for the pharmaceutical company to consider further investment in this area, which is disappointing to me as a patient, as I did feel a lot better when having the ‘mystery liquid’.
I wrote about the role of medical research, here
As a rare disease patient, this (taking part in trials), is a way in which we can help to pioneer the future for fellow rare disease patients.
As well as, by taking part in a trial there is the opportunity to try newer innovative treatments which may provide most welcome symptom relief.
Raremark website here
To read my articles:
Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here
March Autoimmune Disease Awareness Month 2018, Click here
Global patient video, Click here
Becoming a Patient Research Ambassador for the NIHR, Click here
If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here
Expert Specialist Centres, Click here
World Scleroderma Day 29th June 2016, Click here
World Scleroderma Day 29th June 2017, Click here
Why Global Collaboration is important to the Rare Disease Patient, Click here
2016 Rare Disease Day Patient Voice