Friday, 2 June 2017

Why Research and Patient Profiles? Scleroderma Awareness Month 2017, Raynaud's, Rare Autoimmune Disease


1st June, saw the start of Scleroderma Awareness Month, with the 29th, being World Scleroderma Day. 

Further to this year’s Rare Disease Day 2017 theme being ‘Research – With Research Possibilities are Limitless’, I have made ‘Research’ the theme of my Scleroderma Awareness Month 2017 Campaign, along with patient profiles.   

To read my articles for Rare Disease Day 2017: 

'Medical Research and its importance', Click here 

'Clinical Unmet Needs for the Raynaud's Scleroderma patient', Click here

In this article, I intend on discussing the importance of research, combined with a global collaborative approach. 

As well as, how raising scleroderma awareness during June, can maximise the potential of the cause and cure of this rare disease being discovered, in our lifetime. 

I discussed more about how research provides immense hope to the scleroderma Raynaud’s rare disease patient in my last article about the importance of scleroderma awareness month, Click here  


Why patient profiles?  

This autumn, 2017, marks my 20th anniversary of living with the disease. 

Upon my initial diagnosis, in 1997, aged 24, I was given a prognosis of 15 months.

At that time, I was working as a medical representative for Bristol Myers Squibb, as well as, studying on a part time distance learning basis to achieve my dream to become a barrister. 

I consider myself to be an extremely ‘lucky’ scleroderma Raynaud’s patient, in that, I have minimal internal organ damage. 

I contribute this to the excellent care which I received from having attended the Scleroderma Unit at the Royal Free Hospital, London, December 1998, and, I continue to attend. 

Within 30 seconds of my appointment with Dame Professor Carol Black, I knew that she would use all of her medical expertise to help me get better and help me realise my dream. 

Several years of intense chemotherapy infusions combined with the immuno-suppressant, mycophenolate, eventually stabalised what was a very aggressive diffuse form of the disease. 

I was also on the stem cell transplant waiting list.  

By now, my hero, Professor Chris Denton had become my lead Doctor, and to whom I am eternally grateful to, and will forever remain so. His dedication and commitment to his patients and the scleroderma Raynaud’s global community is superhuman.   

Prof Denton helped me realise my dream. 1st March 2004 I qualified as a self employed practising barrister in my own right. 

Sadly, if you have followed my blog, you will know that my ‘tin man’ body due to the damage from scleroderma, combined with my extreme Raynaud’s sensitivity, makes being ‘under house arrest’, my optimum symptom management location, and the internet my lifeline. 

Read more, here  

I therefore feel ‘lucky’ that I had, in effect, an early diagnosis whereby treatments still remained an option to me. 

Even though managing my symptoms, combined with the damage to my body caused by the scleroderma, makes for a full time job. 

Any spare energy or time which I have, is dedicated to the scleroderma Raynaud’s cause. 

To read my article ‘Ah great, another day to spend in bed, said no scleroderma Raynaud’s rare disease patient’, Click here 

I have a new dream now – a Scleroderma Free World, and a Raynaud’s Free World.   

I very much hope that this dream becomes a reality in my lifetime. 

I believe that it will, upon investment and global collaboration in research, not just for scleroderma but for the autoimmune diseases as a whole.  

Further to my personal experience, and my new dream, I am passionate that a scleroderma Raynaud’s diagnosis is not subject to a postcode / ZIP / Country lottery, as to best practice and care for the patient.  

Along with, expert specialist centres being essential, and medical research being a priority. 

The Scleroderma Unit at the Royal Free Hospital is the UK national scleroderma clinical trials centre.   

Due to scleroderma being a rare disease, and therefore having low patient numbers, clinical trial recruitment is a challenge. 

It is difficult to obtain clinically sound, scientific statistically significant data, with only a small population eligible to take part. 

This is why global collaboration is crucial for the sharing of best practice for the scleroderma Raynaud’s patient.  

Taking part in a clinical trial is an opportunity to have access to the most advanced potential therapies. Read more about my clinical trial experience, here  

During the lead up to June, I contacted several patients from around the globe, asking for them to complete a short questionnaire. 

I designed the questionnaire to highlight the global diagnostic approach, as well as, treatment for the global scleroderma Raynaud’s patient, and how their experience could be improved.  

I am truly humbled to all those who took part and thank each and every patient who shared their reality, to help put the spotlight on this life changing, if not life threatening, rare disease. 

Follow the campaign at Scleroderma News, as well as, my Raynauds Scleroderma Awareness Global Patients Facebook Page 

Living the dream, scleroderma style, hoping for a cure

#SclerodermaFreeWorld #RaynaudsFreeWorld  

Please donate to my Just Giving page for the Scleroderma Unit at the Royal Free Hospital where 100% of your donation will be used for medical research only. Thank You. 




An edited version of this article was published here, in my Column with Scleroderma News. 
June 2017

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      

If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here   


Scleroderma Awareness Month 2017 Patient Profiles Campaign, Click here     
 
Scleroderma Awareness Month 2017 Patient Profiles Campaign Patient Index, Click here        
The Pandora’s Box of the rare autoimmune disease Scleroderma, Raynaud's and Cancer, Click here



Key Event Dates 2018, Click here     

Importance of an early diagnosis, Click here     

Taking Part in Clinical Research Trials, Click here    


The Importance of a Multi Disciplinary Medical ‘Dream Team’, Click here 

Expert Specialist Centres, Click here      

My Skin is Cured from Scleroderma, Click here      

UK Guidelines for Managing and Treating Scleroderma, Click here      

Fatigue, Click here      

Mobility, Click here       


Raynaud's, 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      



RARE DISEASE DAY:
The theme to this years Rare Disease Day is Research

Rare Disease Day 2018 – Research, Taking Part in Clinical Trials. Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here     

2016 Rare Disease Day Patient Voice
2016 Rare Disease UK Parliamentary Reception 

2017 Rare Disease Day Flashback  

More unmet clinical needs  

Rare Disease Day 2016



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. 

I highlighted other areas of medical research interest within Week 3 of my Patient Profiles Campaign for Scleroderma Awareness Month 2017. 

VIDEO



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: 

Early Diagnosis
Expert Specialist Centres
Access to Innovative Medicines

Where MEDICAL RESEARCH investment is VITAL.

Research is the key. Abstracts from 2016 World Congress, Click here


Scleroderma Awareness Month Campaign 2016, Click here

View video, here

Preamble - here 



The Family Day at the Scleroderma Unit, The Royal Free Hospital is taking place on the 19th May 2018. Browse the program, here

This year, I am celebrating 20 years of being a patient at this world leading expert specialist research centre. 

Sept 2017


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.
I am truly humbled and inspired by their work ethic. 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 diagnosing doctor, that I was looking at a 15month prognosis. 

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.
  
For latest updates follow: 

Facebook Page:

Twitter: @SclerodermaRF  @RaynaudsRf  


#SclerodermaFreeWorld           #RaynaudsFreeWorld
#ADAM #Scleroderma
#Raynauds
#RareDisease  

Living the dream, scleroderma style.  


Please DONATE to help fund medical research at The Scleroderma Unit,The Royal Free Hospital, London.  



100% of your monies will be used for medical research purposes only. Thank You. 
#HOPE 


 

Last Update: April 2018




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