Rare
Disease Day 2018: Research, Taking Part In Clinical Trials.
Scleroderma,
Raynaud's, Autoimmune Rare Disease.
#RaynaudsFreeWorld
#SclerodermaFreeWorld
Today, February 28th,
is Rare Disease Day.
This year’s theme is Research.
Living with a rare disease is daunting in every way imaginable. Having been a diffuse scleroderma, rare disease patient for 21 years, in my view, Research provides immense hope.
Living with a rare disease is daunting in every way imaginable. Having been a diffuse scleroderma, rare disease patient for 21 years, in my view, Research provides immense hope.
Hope that the desperately
needed understanding as to cause and cure will be discovered. With an
improvement in medicines, being a soother in the meantime.
So, what is a rare disease?
A
disease or disorder is defined as rare in Europe when it affects fewer than 1
in 2000.
A disease or disorder is defined
as rare in the USA when it affects fewer than 200,000 Americans at any given
time.
There are 2.5 million scleroderma patients
worldwide, as by The World Scleroderma Foundation
The Role of Medical
Research to the Rare Disease Patient, plays a pivotal role in improving the rare disease patient experience.
This year’s theme of Research focusses on how important the
patient is, to the entire medical research arena.
Including, clinical trials,
from the design to the actual live participation.
During the course of the last
few decades, I have taken part in several clinical research trials.
I am not the most willing of
participants for taking any kind of drug, especially not licensed drugs.
However, for medical research clinical trial purposes, all ‘mystery’
medications have undergone rigorous testing, prior to reaching the clinical
trial stage of testing on humans. Although, this does not eliminate every or
any potential risk of taking the drug.
For a new medication to reach
the market place, strict criteria must be met for the medication to become
licensed for the indications which it represents. This is where the design of
the clinical trial is imperative with its detail.
In 2015 I became a patient
expert adviser with The European Medicines Agency.
I very much enjoy this
role
I have taken part in
various Scientific Advice Working Party
SAWP and Committee for Orphan Medicinal Products
COMP meetings.
It is very exciting for me to
see in this setting, the increase in pharmaceutical interest for scleroderma
patients.
I have been involved with many
clinical trial design set ups with chemical substances which only have a
number, and have not even been named yet.
In relation to the clinical
trial design for scleroderma patients- ALL clinical trial assessment criteria
now includes digital ulcer involvement, further to my insistence!
I am delighted to have recently become a Patient Research Ambassador for the Dermatology
and Musculoskeletal departments at The Royal Free Hospital.
There are many clinical trials currently taking place at the Scleroderma Unit.
The
Christmas 2017 newsletter
gives a summary of Research progress so far at the Scleroderma Unit.
All of the trials which I have participated in, have been conducted at the Unit.
All of the trials which I have participated in, have been conducted at the Unit.
Sadly,
there remains many unmet clinical needs to the scleroderma patient.
The Pandora’s Box continues to present challenging surprises, making my symptom management a full time job.
This
year’s Scleroderma Family Day is to be held on Saturday 19th May in
the atrium at the Royal Free Hospital. To view the program, click here.
I am
very much hoping to attend this year's family day, as I am celebrating my 20th
year anniversary of being a patient at the Unit.
This Scleroderma Olympian is already in training, currently in full time hibernation.
This Scleroderma Olympian is already in training, currently in full time hibernation.
Unfortunately,
I will not be attending the Rare Disease UK Parliamentary Reception, this year.
However the 2016 parliamentary reception still remains one of my few patient highlights.
However the 2016 parliamentary reception still remains one of my few patient highlights.
I discussed other areas of
medical research interest within Week3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017, Flashback.
As
well as, highlighting the need for RESEARCH investment.
The 'rare disease' label can often feel like a
bleak and lonely existence.
Although
rare disease patients are few in number, eg. 2.5 million scleroderma patients
worldwide (as by the World Scleroderma Foundation), the commonalities and
golden hallmark for each rare disease patient are the same:
Where MEDICAL RESEARCH investment is VITAL to
improving the entire rare disease landscape, including, the rare disease
patient's day to day reality.
Thereby, unlocking the rare disease enigma.
Living the dream, scleroderma
style.
Please DONATE
to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London where 100% of all of your monies will be used for medical
research purposes only. Thank You.
Wishing you a very happy Rare
Disease Day, feeling good.
#SclerodermaFreeWorld
#RaynaudsFreeWorld #HOPE
#RareDiseaseDay
An edited version of this article was published here, in my Column with Scleroderma News.
February 2018
Scleroderma Family Day
2020
25th
Anniversary Meeting
The Atrium, Royal Free
Hospital, London, NW3 2QG
Chair: Professor Chris
Denton
PROVISIONAL PROGRAMME
09.30 – 10.00 Registration
and Coffee
10.00 – 10.20 Welcome Dame
Carol Black
Prof Chris Denton &
David Abraham
10.20 – 10.40 What
is a Biopsy? Dr Kristina Clark
10.40 – 11.05 Dental
aspects of Scleroderma Prof Stephen Porter
11.05 – 11.25 Gastrointestinal
problems – Dr Fiza
Ahmed
shedding
new light on old problems
11.25 – 11.50 Pulmonary
hypertension and the heart Dr Gerry
Coghlan
11.50 – 12.15 Scleroderma
cohort studies – Dr
Francesco
‘Learning
from our patients’ del Galdo
12.15 – 14.15 LUNCH
BREAK – see below
14.15 – 14.45 25
years of progress – Prof
Chris Denton
from
‘black box’ to ‘positive trials’
14.45 – 15.15 International
speaker – Dr
Madelon Vonk
Scleroderma
Management in Netherlands
15.30 Raffle
Lunchtime Discussions Groups /
Demonstrations include:
Clinical Trials Rachel
Ochiel and team
National Institute of Health Research Christine Menzies
Drug Information / monitoring Pharmacy
Massage Keith Hunt MBE
Pulmonary Hypertension Education / nursing Sally
Reddecliffe/Adele Dawson
Rheumatology Laboratory Research
Laboratory Staff
Scleroderma Education / Nursing Louise
Parker/ Joseph Cainap
Thermography Dr Kevin Howell
SRUK Ollie Scott
Sjogren’s syndrome British
Sjogren’s Society
To Read My Articles:
Gift in My Will, Click here
Planning for the Future, Click here
Rare Disease Day:
Rare Disease Day 2020:
Listen to my interview with John Smeeton, (Silver Fox), from the Royal Free Radio, here
Raise awareness and donate to medical research, order your #SclerodermaFreeWorld #RaynaudsFreeWorld tshirt here
Rare Disease Day 2020:
Listen to my interview with John Smeeton, (Silver Fox), from the Royal Free Radio, here
Raise awareness and donate to medical research, order your #SclerodermaFreeWorld #RaynaudsFreeWorld tshirt here
Rare Disease Day 2019: 11am 97.4RockFM headlines, NO CURE, Click here
2016 Rare Disease UK Parliamentary Reception
Rare
Disease Day is a fantastic opportunity for the entire rare disease
community to shine a spotlight on their reality, combining as one unified
voice. Where, at least one commonality presides –
Medical
Research
provides the brightest light,
for the illumination of
the rare disease patients’ plight.
provides the brightest light,
for the illumination of
the rare disease patients’ plight.
Although
rare disease patients are few in number, eg. 2.5 million scleroderma patients
worldwide, (the World Scleroderma Foundation), the commonalities and golden
hallmark for each rare disease patient are the same overall.
To read my articles:
Celebrating 20 years of being a patient at the Scleroderma Unit, Click here
2019 New Challenges, Click here
NIHR Video: 'My
Experience of Clinical Trials', Click here
If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here
The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here
If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here
The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here
2018 Scleroderma Awareness Raising and Medical Research, Click here
SCLERODERMA:
Global Patient Profiles 2018 Video, Click here
Unmet Medical Needs, Click here
Calcinosis Video, Click here
The scleroderma tooth fairy, Click here
Skin Cancer and scleroderma, Click here
Unmet Medical Needs, Click here
Calcinosis Video, Click here
The scleroderma tooth fairy, Click here
Skin Cancer and scleroderma, Click here
 |
| Sept 2017 |
 |
| Prof Chris Denton and I, Sept 2017 |
 |
| June 2019 |
 |
I am eternally grateful to the global scleroderma trail blazers Dame
Prof Black and Prof Chris Denton, whose commitment and dedication to unlocking
the scleroderma enigma, is nothing other than, superhuman. Along with the
Raynaud's world trail blazer, Dr Kevin Howell.
I am truly humbled and inspired by their work ethic and commitment to
their patients.
I am wholly appreciative for Prof Denton’s continued medical expertise
and support, especially during my barrister qualifying years, 1997 -
2004.
1st March 2004, I qualified as a self employed practising
barrister. Further to having been told in 1997, by my original diagnosing doctor, that I
was looking at a 15month prognosis.
 |
| Chat Magazine May 2019 |
I very much hope to utilise my professional skills
and qualifications along with my patient experience, to help achieve the
#SclerodermaFreeWorld dream, hoping to improve understanding and best practice,
in the meantime. Read more, here
World Scleroderma Day
2015, 29th June.
 |
| James Carver, myself, Prof Chris Denton |
I had the pleasure of being a presenter and part of
the European co-hort who gave a presentation at the European Parliament,
Brussels, in honour of World Scleroderma Day 2015.
I am immensely grateful to James Carver, former MEP, for organizing this event in memory of his late wife Carmen, who sadly passed from Scleroderma.
I am immensely grateful to James Carver, former MEP, for organizing this event in memory of his late wife Carmen, who sadly passed from Scleroderma.
To view the presentation, Click here
Raynaud's:
October:
Raynaud's, Click here
 |
| The global Raynaud's trailblazer - Dr Howell and I, Sept 2017 |
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100% of your monies will be used for medical research purposes only. NO wages or admin costs. Thank You.
Last Update: Feb 2020.
Rare
Disease Day 2018: Research, Taking Part In Clinical Trials.
Scleroderma,
Raynaud's, Autoimmune Rare Disease.
#RaynaudsFreeWorld
#SclerodermaFreeWorld
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