Saturday, 24 June 2017

Week 3, Medical research areas of current interest, Scleroderma Awareness Month, Raynaud's, Rare Disease 2017, Patient Profiles Campaign


Week 3 – Scleroderma Awareness Month 2017, 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 RareDisease 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 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 15month prognosis.   

An edited version of this article appeared here in my Column  with Scleroderma News  

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       

Week 1 Scleroderma Awareness Month, Raynaud's, Rare Disease, 2017 – Patient Profiles Campaign, Click here

Week 2 Scleroderma Awareness Month, Raynaud's, Rare Disease, Patient Profiles 2017, Progress in medical research, Click here

2017 INDEX Scleroderma Awareness Campaign Patient Profiles Click here      

INDEX to Scleroderma Awareness 2016 Campaign, Click here 

To read my articles  

Importance of an early diagnosis, Click here 

Taking Part in Clinical Research Trials, Click here

Expert Specialist Centres, Click here 

My Skin is Cured from Scleroderma, Click here 

UK Guidelines for Managing and Treating Scleroderma, Click here

World Scleroderma Day 29th June Click here    

Please donate to help fund medical research at The Scleroderma Unit where 100% of all of your monies will be used for medical research purposes only. Thank You.
June 2017 Total

Living the dream hoping for a cure

#SclerodermaFreeWorld #RaynaudsFreeWorld 

#RareDisease #Hope #Belief 

Follow Raynauds Scleroderma Awareness Global Patients Facebook Page for latest updates   

Thanks to USA patient Sharon Esposito for sharing her wonderful creativity

Please use the icons below to share this article #SclerodermaAwareness  

#SclerodermaFreeWorld #RaynaudsFreeWorld
#RareDisease #Hope #Belief

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