Saturday, 24 June 2017

Medical Research Areas Of Current Interest. Week 3, Scleroderma Awareness Month 2017, Raynaud's, Autoimmune Rare Disease. Patient Profiles.

Medical Research Areas Of Current Interest

Week 3: Scleroderma Awareness Month 2017

Raynaud's, Autoimmune Rare Disease. Patient Profiles
   
Medical Research Areas Of Current Interest

Week 3: Scleroderma Awareness Month 2017

Raynaud's, Autoimmune Rare Disease. Patient Profiles

Medical Research Areas Of Current Interest  

The month of June is Scleroderma Awareness Month, with 29th June, being World Scleroderma Day.  

Further to the theme for Rare Disease Day 2017, being ‘Research, Possibilities are Limitless’, I have made ‘Research’ the theme of my Scleroderma Awareness Month 2017 campaign, along with patient profiles from various countries around the world, highlighting the crucial need for further investment in medical research, in patient unmet clinical needs.  

I described in my article for Week 2, my personal drive for global ‘best management and treatment guidelines’. 

Where without doubt, the global golden hallmarks for such are:  

Early diagnosis  

Expert specialist centres  

Access to innovative medicines  


So far, all of the patients included within the patient profile campaign, highlight the crucial necessity for these 3 hallmarks to best practice. 

As well as, the patients show the unmet clinical needs, where the medical research is slowly progressing.  

In this article, I intend on discussing the most up to date medical research understanding, as to the many different aspects, to the scleroderma and Raynaud’s, Autoimmune Rare Disease.  

At the 4th Systemic Sclerosis World Congress held in Lisbon 2016, The Journal of Scleroderma and Related Disorders was launched. 

This is the ‘Official Journal of World Scleroderma Foundation and the European Scleroderma Trials and Research group (EUSTAR)’.      



Included within Volume 1 Number 1 January – April 2016 issue are Abstracts which were presented at the Congress. 

I have made a summary of the subject headings below, as well as, I have summarised each Abstract Content, Click here


Session 1:     Pulmonary involvement  

Session 2:     Ulcers  

Lecture 1:      Macitentan responsiveness supports the validity of a murine model of pulmonary hypertension in scleroderma associated with altered TGFBETA/BMPRII signalling. 

  

Parallel Session 3:   Common clinical situations with poor evidence based data: How to manage?   

Parallel Session 4:   Advances in epigenetics and genetics   

Parallel Session 5:   Cardiac   

Parallel Session 6:   Current research in immunity / inflammation
   

Lecture 3:   Increased frequency of malignancies, and in particular breast cancer, synchronous to the onset of SSc in anti-RNA polymerase III antibodies positive patients: A EUSTAR multicentre study.
    

Lecture 4:      Impaired Micronutrient Status In Patients With SSc  
  

Session 8 :    Round table SSc registries in the world   
  


Lecture 5:      Survival and organ involvement in patients with limited cutaneous SSc
 and anti-topoisomerase antibodies: more like lcSSc or dcSSc?    

Parallel Session 9:              Experimental Models of Scleroderma    

Parallel Session 10:            Outcome Measures 
     

Session 11:   Emerging therapies   

   
Although the discovery of a cure is unlikely to be of huge assistance to me, since my disease level is stable, the subject contents of the Abstracts listed above, are most encouraging. 

They show that an improvement in understanding of all aspects of the disease are being looked into, a ‘holistic approach’.   

On a personal level, it is most rewarding for me to see some of the work which I am involved with, via my role with the European Medicines Agency, being utilised into the progression of improving the SclerodermaFreeWorld goal. 

I am honoured to be a member of the Scientific Advice Working Party at the EMA, where pharmaceutical companies and the like, for example, seek advice as to how to progress a clinical trial drug for purposes of finally being accepted into the marketplace as a licensed brand.   

I took the liberty to use DAY 12 of the patient profile campaign to highlight my experience, as this was my 44th birthday. I had the most perfect day – huge thanks to those who made it so special.


My 44th birthday, having been diagnosed with systemic sclerosis aged 24, and given a 15month prognosis

The 5th Systemic Sclerosis World Congress is due to take place Feb 15 – 17  2018 in Bordeaux, France next year. 

This bi-annual event is the hub of the most up to date understanding of the entire scleroderma disease process.   



Overall, an improvement in the medical understanding as to the nature of the disease has improved immensely over the course of the last decade. 

However, due to the multi complex body systems involved in the disease process, research still falls way behind with providing an understanding as to the cause, and effective treatments, leading to a cure.   



The countdown is on… 29th June, World Scleroderma Day. I will be presenting live on the Scleroderma News Facebook page, Please Join Me, and please post any questions / comments which you would like to be discussed. 

In particular, I will be focussing on this years awareness campaign, as well as my 20th year anniversary of living with scleroderma and Raynaud’s, having been given a 15 month prognosis.  

To read more about why I chose ‘Patient Profiling and Research’ as the focus of my 2017 campaign, Click here    

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

2017 INDEX Scleroderma Awareness Campaign Patient Profiles, Click here    


INDEX to Scleroderma Awareness 2016 Campaign, Click here     




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   

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   


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    


Diet and nutrition, 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  
  


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

Links

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. No wages or admin costs. Thank You.
#HOPE 


 

Last Update: April 2018    

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