2019 more answers to questions

Article No 306

20 December 2019

If you have genetic mutations in your Complement and/or  in related components in your blood which cross talk and hamper Complement’s ability to regulate itself ; and if  your TMA has been triggered by a kidney transplant or pregnancy or  surgery or drugs  or something yet unexplained , and you  need a Complement inhibitor, aHUS alliance Global Action will be searching to bring you more information.
The aHUS alliance Global Action’s website has become one of the most active, inclusive and up to the minute aHUS sources online. Written by those affected by aHUS  for those affected by aHUS.
What matters to aHUS patients and their families underpins everything that the patient groups that have joined together in the alliance do.
The research agenda created by aHUS patients and families for aHUS patients and families tells us what matters.
Officially  adopted by the alliance on Rare Disease Day, 28 February 2019 with the message “you are not alone, we can find a way”, during 2019 the aHUS alliance Global Action website has featured articles related to the topics in your Agenda.
Below is a compendium of those 2019  articles  indexed by the topics in the Agenda.

  1. aHUS Causes and precautions

1.1 Are the predisposing genetic and triggering factors of aHUS fully catalogued and understood and will it help to know how variable are the risks of these between individuals?
Complement database leader interview
A well of aHUS mutations
Anti-FHs aHUS -a Disease within a Disease
aHUS Trigger- S. Pneumoniae
aHUS is its end really nigh?
1.2 When it comes to genetic testing of aHUS family members what is best – to know or not to know -and what can be done with the knowledge?
aHUS genetic counselling – evidence based

  1. aHUS Diagnosis

2.1 Is there a diagnosis sweet spot which can be found before a developing thrombotic microangiopathy turns into a catastrophic episode of aHUS?

  1. aHUS Treatment

3.1 Is there an optimal way in which a complement inhibitor can be delivered to suit an individual’s need?
A Seminal Article on Global aHUS 2019
Let the science give the answers
3.2 Can a clinically effective therapy be developed that is affordable for all aHUS patients?
10/18/19- FDA Decide
Small Molecule Factor D Inhibitors – Right for aHUS
Ravulizumab for aHUS
Generium’s: Elizaria: First Biosimilar to Eculizumab
aHUS Trials Watch 8
aHUS Trials Watch 7
3.3 Is it more cost effective, as well as clinically effective, for the management of an aHUS patient’s treatment to undertake genetic testing?
ASN aHUS and the sisters from Missouri
3.4 Can a complement inhibitor be stopped safely when not needed by some aHUS patients and what makes them different?
3.5 Is there a significant difference in outcome between having a complement inhibitor before or after a kidney transplant?
Transplants , aHUS and eculizumab
ahUS Transplants – Abstract video
3.6 Will there be a cure eventually?

  1. aHUS Impact – clinical/psychological

4.1 Are there long-term studies of outcomes of those in remission whether treated by a complement inhibitor or not?
Remission from aHUS
4.2 Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?
Eculizumab Safety
Eculizumab -safe or not
aHUS Fatigue it matters to aHUS patients
CardioRenal Syndrome:The Relationship of Heart & Kidney Health
Dialysis Diaries – long term complications
Dialysis Diaries – heart problems
Some side effect – meningococcal infection 
An aHUS Tale from Slovakia 
4.3 Does the anxiety and self-esteem of aHUS patients vary significantly between treatment types and what can be done to reduce and boost them respectively?
The role of Positive Health in the Dutch approach to aHUS
Finding the new normal after diagnosis
The Zen of living with aHUS
5.aHUS Impact- Socio-Economic
5.1 How does living with aHUS impact on education and work?
An aHUS Patient Entrepreneur
“Away from home” Eculizumab infusions
About aHUS: for Youth Leaders, Camps & Coaches
5.2 Do aHUS families have all the correct information to make informed family planning decisions?
The right choice- Pregnancy and aHUS
5.3 How many aHUS patients are there in my local area, my country and the world and how do they differ?
That is it for 2019.
aHUS patients everywhere frequently share information about themselves to each other. Facebook is full of comments  to help understand more about aHUS but because of its transitory or closed  nature those insights are lost to help the global community.
As well as getting answers, those affected by aHUS can volunteer important insights  which can be harnessed for the greater good.
Something to think about for 2020.

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