65 questions, 29 article blogs with answers to the questions published between 7 March and 16 May and over 300 views of the video aHUS alliance Global Action fulfilled its intention to keep the Rare Disease Day 2022 video alive long beyond Rare Disease Day itself.
There have been nearly 3000 views of the blogs to date but there will be many more in the future, when the articles appear in internet search results for the topic in the question.
But it does not stop there.
The blog article introducing the series showed how the video questions fitted into the aHUS Patients Research Agenda. They did and the agenda held up well.
However it was noticeable that priorities were shifting. Also there were questions which added new slants to the original scope of the original agenda.
A couple of examples to illustrate.
Firstly a question from Sebastian about aHUS and recreation. It is saying that there is more to life than work or education. Sports participation is important too. So adding “recreation” to ” How does living with aHUS impact on education and work?” makes the socioeconomic aspect of aHUS more complete.
Another from Enzo is about adverse side effects but adds the dimension of time. So it is not just what is experienced by patients here and now but what cumulative effects may emerge.
Now that all the replies are completed, time will be spent in looking at some revisions to the wording of the research agenda so it keeps relevant to what matters to aHUS patients.
Global Action will report back later this year.
Article No. 516
All the articles links are together below.
A final question in the RDD2022 video series on how to integrate a patient voice of experience into aHUS Pharma and aHUS conferences. Patient centricity is now a byword for…CONTINUE READINGIntegrating a greater aHUS patient voice
This question about difference in disease triggers and treatments from Jeff did not feature in the RDD2022 video but was given as example in the invitation to participate in the…CONTINUE READINGDifference in aHUSs
A number of questions in the RDD2022 video are about what will happen to patients in the future due to the disease or eculizumab. Predicting the future is tricky. It…CONTINUE READINGFuture effects of aHUS and its treatment
The care pathway for aHUS patients has been looked at in detail in Global Action’s Diagnosis Process Study ( for reports published to date click HERE ). None of the…CONTINUE READINGaHUS care pathway
Zinovieva asked two related questions in the RDD 2020 video The issues of what provokes ( or triggers) the development of aHUS or starts it again, relapse , after being…CONTINUE READINGaHUS limitations
One thing is for certain, there is much more information available about aHUS because of the internet than there was even 10 years ago. There are so many facets and…CONTINUE READINGaHUS Patient information and mutual support
Usually inheritance questions about a HUS relate to genetic susceptibility mutations/variants/markers that are known. This one is different because it is about patients who are idiopathic ( cause is unknown)…CONTINUE READING
There was one question about COVID vaccinations and aHUS in the RDD2022 Video, Global Action featured several articles about COVID vaccination including advice given by clinicians as well as the…CONTINUE READINGWhat about aHUS and COVID Vaccinations and access to genetic testing and genetic research
The RDD2022 Video question by Heitor may be one which is not specific to aHUS, . It raises issues, which are common to all affected by life threatening diseases, no…CONTINUE READINGaHUS survival and social support
Most babies are born healthy. Even those susceptible to aHUS due to genetic abnormalities are born healthy. Although it is rare, it has been known for newborns to onset within…CONTINUE READINGScreening for aHUS at birthSymposium on aHUS in 2022
The twice delayed ISN Frontiers Symposium on Complement related renal diseases – MPGN/C3G and aHUS is still on course to be held in Bergamo, Italy on 23rd to 25th June…CONTINUE READINGSymposium on aHUS in 2022
Pregnancy is an important topic and has featured a lot on this website. Quite a few questions about it in the RDD2022 video though. A lot ore is known about…CONTINUE READINGCan aHUS relapse in pregnancy?
Three questions in the RDD22 video are each very specific but reflect the diverse impact of having had aHUS. Firstly one about Strokes and aHUS. A stroke occurs when blood…CONTINUE READINGStrokes, DGKE , Sports & Recreation and aHUS questions
It is known that certain rare genetic variants, mutations, or markers, in components of the Complement System make some people susceptible to aHUS. it is less known that there are…CONTINUE READINGQuestions about aHUS triggers
Although aHUS has genetic causes, in the majority of cases it is found that the patient is the only one in the family with the genetic susceptibility for aHUS. That…CONTINUE READING
Questions about hormones and aHUS in the RDD2022 video. It is a feature of aHUS that it becomes more common in females after puberty and during pregnancy. Both puberty and…CONTINUE READINGHormones and aHUS
Remission from aHUS is different from having been cured because there is still an uncertainty that aHUS can return. If that happens it is called a relapse. For a relapse…CONTINUE READINGaHUS remission chance and relapse risk
Another question from the RDD2022 video. This one from Harry in Hartlepool UK. This one is about anti factor h autoantibodies. It is not known how many cases of aHUS…CONTINUE READINGAutoantibodies cause aHUS
A top topic if the number of questions relating to this issue in the RDD2022 Video are to go by. Drug developments are very important, most importantly to provide more…CONTINUE READINGAre there alternative aHUS drugs and ways to deliver them?
Answers to the RDD2022 video continue. Samantha asks will there ever be a cure for aHUS? Crikey that is a difficult one to answer. The simplest answer is “I don’t…CONTINUE READINGWill there be a cure for aHUS one day?
A kidney transplant used to be contraindicated for those predisposed to aHUS. Although it could have been successful for some depending on their genetic susceptibility, most would lose their kidney…CONTINUE READINGCan a kidney transplant cure aHUS?
Long term aHUS disease, diagnosis and treatment legacies on both physical and mental health have featured in two aHUS Patient Research Agenda topics. The damage to the body during an…CONTINUE READINGLong term aHUS effects
Eculizumab was approved by the US Food and Drugs Administration for PNH for 15 years ago on 16 March. This September coming, it will be 11 years since the FDA…CONTINUE READING
A very important topic for many aHUS patients around the world with an unmet need. Patients have included the topic in their Research Agenda and thought it important enough to…CONTINUE READINGCan aHUS treatment be affordable for all?
Let us kick off the responses to the RDD2022 Video with a couple of questions about the characteristics of aHUS patients. I may have written in the past that if…CONTINUE READINGWhat does an aHUS patient look like
This article marks the 500th website news “blog” since it went live nearly six years ago. That milestone is marked by an article on questions from the in RDD2022 video…CONTINUE READINGMonitoring after Eculizumab withdrawal
Ravulizumab is eculizumab, Carsten. They work the same way. It is the same monoclonal antibody. It has just been modified in a couple of places so that it is effective…CONTINUE READING
Ravulizumab is more widely used these days, particularly in the PNH community. Apart from the longer infusion intervals PNH patients benefit from higher dose levels that stop breakthrough haemolysis which,…CONTINUE READING
When Jeff, the producer of these awareness day videos, suggested that “questions” should be the theme of this years’ RDD Video I was surprised. Surprised because it had been five…CONTINUE READING