Conversations tagged muckle-wells

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  alicat2011 | Medication | published about 1 year ago | Originally written in English Canakinumab (Ilaris) muckle wells

  Hi there.
  My name is Alison and I would love to here from who is on Canakinumab (Ilaris) I'm on it, and next month will be my 5th dose. Also, if you have Muckle Wells, how has is your life improved?

  I really suffered with the rash, sore eyes and joint pains which had a major effect on my childhood and now touch wood thanks to Canakinumab (Ilaris) it is kept at bay

  My 3 year old child also suffers and I am thinking about once she turns 4, starting her on treatment that way she will have a better childhood hopefully.

  Reply to me with your thoughts...
  27 replies to the topic — see all replies

  • 
    karend published about 1 month ago | Originally written in English

    Dear Anna,

    Have you had any success with getting your daughter tested for CAPS, or have you been able to get her the medication? There is another family that has just joined this group that is from Russia, and it would be nice to find out if you have had any success with getting treatment for your daughter.

    Thanks,

    Karen Durrant
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    AnnaKolev published about 1 month ago | Originally written in English

    Dear Karen
    I know - she has already called me. We have sent uor blood tests to NIH USA and after 1-2 weeks the result will be ready. I hope that they will advice me therapy ( if anakinra with which dose it;s better to start with maybe ones a week or I know that in USA be tablet therapy ( asatioprin sometimes ) can be used. By the way we are preparing all document to ministry of health to get permissin to supply anakinra from abroad for our children. I also have seen about study programmes for Ilaris - but they send me to Moscow office - where on information. How much Ilaris cost? Just to understand if our government will manage to buy it for us ? Anna
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    karend published about 1 month ago | Originally written in English

    Dear Anna,

    I am very glad that the doctors at the NIH are helping you! This is good news. I had contacted them, but had not heard what had been done. It is also very good that you are working on trying to get permission from the government to have anakinra for your children. If you need help with this in any way, please let me know. You can email me directly at: karen.nomidalliance.org@gmail.com too. I have some forms that we can use to try to apply for the medication coverage, and I can try to help contact the drug company, but we will need to wait until your genetic testing is back. Your work with the government to get approval for this drug to be brought into Russia will be very helpful for your child, and many others too. Thank you!

    I am very glad that you are also talking with the other family from Russia on this group! I am hopeful that you have also been put in touch with the other 2 families in Russia that are getting help for their children that have CAPS too.

    As for the cost of Ilaris, it is very expensive, but in countries where the drug is approved, the insurance covers most of the cost, and there are patient assistance programs to also lower the cost to patients in the US. Anakinra is not as expensive, but it still costs a lot of money. In America, a month supply of anakinra based on 1 syringe a day of medication may cost around $20,000 a year, but Ilaris averages around $100,000 a year at the standard dosing. For some patients, they require higher doses of these medications. Here is an article about these drugs,a nd cost fro m2011: findarticles.com/p/articles/mi_hb4393/is_1...

    Anakinra works out to be around $1600 for 28 syringes (4 weeks supply). For a young child, this is more than enough medication to cover their needs, even if they need a higher dose, but as they get older, they will need a higher dose..

    One 150 mg vial of Ilaris costs around $16,000 but lasts for around 8 weeks for most patients, so it would cost around $8,000 a month, if you were getting Ilaris dosing every 8 weeks, or every 2 months (6 doses a year). Some patients need a much higher dosing of that medication, so the cost would be higher.

    So Ilaris costs about 8 times MORE money than the anakinra per year. Anakinra is a daily injection. Both drugs work very well for patients, so I am hoping that your government will approve one of them to help your child.

    Ilaris is approved by the European Medicines Agency for CAPS, but at this time anakinra is prescribed for CAPS as an off label prescription, which means that the drug is not officially approved at this time for CAPS by the medical agencies, as the drug was originally designed and approved for Rheumatoid Arthritis.

    I hope this helps!

    Karen
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  Jennie39939 | Muckle-Wells | published 5 months ago | Originally written in English New, introducing myself

  Hello, my name's Jennifer. I just found/joined this site. I was born with MWS. My mom says I came out covered in hives, but the doctors told her it would go away as I grew up. Wow, well that didn't happen.

  I wasn't PROPERLY diagnosed until I was 20 years old - though I was misdiagnosed dozens of times. Now I'm 26 and on Illaris and doing quite well. I had all the symptoms of MWS and had a hard time growing up, dealing with the pain and fatigue was the hardest - not knowing what was wrong with me. I was in and out of hospitals my whole life. Every time a joint swelled up - my parents and I just assumed I must have hurt myself - what else could it have been? But no - nothing broken or twisted - just some mysterious unexplainable swelling and pain. Have any of you ever waned to punch your doctors? LOL. Yeah I got pretty frustrated and discouraged at times.

  But like I said, I'm doing good on Illaris. All my symptoms have virtually disappeared and that is a huge relief. I am able to have a mostly normal life now after 20 years. I say MOSTLY normal because I still have a hearing loss that I am still trying to cope with every single day.

  Even though I have the right kind of medication now, I'm tired of feeling "alone" in this. I'm so glad I found this site, I'm actually teary right now. My family has been a great support through and through, especially mom, but I'm a painfully shy person and having to explain it to other people gets old, but I agree we should raise awareness.

  My cousin's little girl may have been diagnosed with CAPS, she had an episode recently. I wasn't able to talk to them about it much because I don't see them a whole lot, but my uncle said "she has what you have". And I feel terrible for that little girl, but I take some small comfort in knowing there is help out there now and she will have a much easier time than I did.

  I truly wish MWS could end with me. But it won't and that's not the right way to deal with it anyway.

  Well, I'll end it here before this post becomes a novel. LOL. Thanks for listening and it's great to meet all of you and read your stories. :)

  ~ Jennifer
  9 replies to the topic — see all replies

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    JacquiAdkins published 5 months ago | Originally written in English

    Wow! Your story almost sounds exactly like mine! I had some of the same issues when I was little and everyone looked at me like I was crazy! I finally got so furious being misdiagnosed numerous times that I did my own research and found the NOMID Alliance page. When I saw the rash picture I screamed out loud "I think I figured it out!!" Well, 2 months later, here I am! I got my first injection of Ilaris today. I was also in tears when I found people that understood what I felt! Because of pages like this I was able to get a diagnosis, and help generations in my family that have dealt with CAPS issues for generations! Good luck with everything! The Facebook page is a great tool to keep in touch as well. :)
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    Jennie39939 published 5 months ago | Originally written in English

    Karen, you're such a blessing putting all these organizations and groups together. I feel like every time I talk to you there is something new to ask my doctor about. :-) I've had MRI scans before but I'm not even sure what they were looking for in them. It was done as part of the study for Illaris. I'm really going to blow my doctor away next time I see her with my new knowledge. It's hard sometimes having to be the one to keep the doctors on their toes, but it's necessary.

    I would love to go to a picnic if I can. I'm sure my family would love it too. Thanks so much for everything and keeping me informed.

    Jennifer
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    karend published 5 months ago | Originally written in English

    Dear Jennifer,

    Thanks to you, and to all the other patients that have joined this forum, and others. With you all, this would not be the same in this community! It is so wonderful to have these ways to connect, and there is so much to be learned about these diseases, and how people live, and thrive with their condition that cannot be found in the medical literature.

    You are too kind! I have to give credit for rarediseasecommunities.org coming about thanks to the hard work of Denis and Rob from EURORDIS, Tai and Mary from NORD, and Paul and the members of AMWS-CINCA in France that were some of the first patients to address the need for such a forum a few years ago, I did get involved in the early phases, and have been pleased to see how this group and the resources have grown to help so many people. I am thrilled to be able to help in any way to get patients, organizations and doctors connected so people can be more informed and empowered patients.

    Paul has been a great inspiration to us in our plans for patient picnics, and I will definitely contact you about that soon. We are in the planning phase and will be sending out information in the next few weeks about proposed places for the picnics, and will be seeking people in various parts of the country to help to get involved in the planning for the events in their area.

    Our org, The NOMID Alliance has learned so much from our work with patients and other organizations, and is here to help you in any way that you need. I appreciate your contributions to this forum, and the ones we have on facebook too. I will be in touch soon, and wish you, and everyone all the best for the New Year!

    Best wishes,

    Karen Durrant
    NOMID Alliance
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  carew52 | Diagnosis | published about 1 year ago | Originally written in English INTRO

  I thought I'd do a little intro, I turn 29 on the 16th of June and currently live in Western Australia. I first learned of Muckle-Wells when my brothers GP first brought it up. Though its really being my sister whose chased up info and got the ball rolling the most. This might have something to do with my darling little niece whose about 18 months old. My sister has since had her blood test as confirmed Muckle-Wells and not just my brother but also my Dad have blood tests pending. There is only me now, having got a referral, who has yet to start the testing process.
  5 replies to the topic — see all replies

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    karend published about 1 year ago | Originally written in English

    Dear Henrik,

    I am also going to send you a more detailed personal email, but want you to know that there are other patients that are very young taking anakinra, and often the dose needs to be raised over a few months to reach the right amount of medication to fully cover all the NOMID/CINCA symptoms. I have heard from a few others that have infants that have experienced the same thing when starting anakinra, There are a few cases that the dosing of anakinra has been split into twice a day to fully cover the symptoms, which has helped, but is not commonly needed with most patients.

    I will contact you via email, and will gladly send you CAPS booklets. Please let me know how many you may need. The NOMID Alliance is a non-profit, so we do not charge for the booklet, but if people want to make a donation to cover costs for shipping, we are always thankful! The CAPS booklet is available online to download for free at any time, until I can mail them to you by post from America. The link is: www.nomidalliance.net/downloads/finalCAPSb...

    Thanks so much for contacting us, and I will reply in more detail to you right away about dosing, and practices with young infants, and also some contacts of specialists that may be able to guide your doctors with this process.

    Best Wishes,

    Karen Durrant

    Thanks,

    Karen Durrant
  • 
    paul published about 1 year ago | Originally written in French

    Pour Henrik

    Bonjour et bienvenu sur le site.

    je confirme les informations de Karen, il y a en France des petits malades qui ont des difficultés pour le traitement. Le spécialiste qui les suit a donc prescrit des dosages spécifiques à raison de DEUX piqûres par jour pour l'ANAKINRA.

    en ce qui concerne ILARIS, je ne connais pas de cas, mais je vais m'informer, ce médicament venant d'être autorisé en France depuis un mois.

    En France aussi, il existe des spécialistes pour le Muckle et Wells Syndrome qui exercent dans des hôpitaux parisiens sous l'appellation "centre de référence"

    ces centres de références regroupent des spécialistes : rhumatologues, pédiatres, ophtalmologues, dermatologues, otorhino-logues...qui travaillent ensemble.

    pour connaitre la maladie MWS, vous pouvez avoir des informations complémentaires sur le site orpha.net.fr qui est disponible en 5 langues.

    vous pouvez aussi bien sûr regarder sur ce site.

    je me présente, je suis Paul Rivière, dont vous trouverez mon profil sur ce site, et suis le Président de l'association Muckle et Wells Syndrome CINCA amws-cinca, et mon parcours du combattant est développé dans la rubrique "comprendre".

    nous venons de mettre en ligne notre propre site (hier soir) www.amws-cinca.eu

    il y a un lien entre ce site et le nôtre : regardez à l'endroit des associations, en dessous de l'association de Karen.

    pour le guides des CAPS, en français, élaboré par le laboratoire NOVARTIS (qui vend Ilaris) et un centre de référence français, le CEREMAI. je vous l'adresse à votre adresse mail

    bien amicalement et au plaisir de vous lire et je souhaite de tout coeur que votre petite fille dispose très rapidement d'un traitement approprié.

    Paul
  • 
    carew52 published about 1 year ago | Originally written in English

    I thought I'd better do an update. I've being approved to try Anakinra and have an appointment scheluded in a couple weeks from now to get started on it.
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  rdc-team | Muckle-Wells | published over 2 years ago | Originally written in English Call for patient testimonies / stories

  There are still many patients living with CAPS in the world who have not yet been diagnosed. We are calling on patients who wish to share their story of diagnosis and living with CAPS to come forward and share your story. It can be in complete anonymity but it will go a long way towards helping others come forward and discuss with their doctor and maybe realise that what they've been living with all these years in fact can be diagnosed and treated.

  If you have lived with Muckle-Wells, FCAS or NOMID and if you'd like to share your story similar to what some other patients have done e.g. bit.ly/paulmucklewells then please message us via the rdc-team profile page (just click on the square logo) or email us at tellmystory.caps@rarediseasecommunities.org
  One reply to the topic — see all replies

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    gilio published over 2 years ago | Originally written in English

    Hello they are the papa of a boy of 17 years, than from 2001 dermatomyositis suffers from, would be a lot lieto to exchange of the opinions with other persons with the same problem, does not know English language but I make translation with web, thanks a lot. Giovanni
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  rdc-team | Medication | published over 2 years ago | Originally written in English Two new treatments with indication for CAPS

  Information relating to two new treatments with an indication for the treatment of CAPS syndromes and which have recently received their Marketing Authorisation from the European Medecines Agency are available at the following URLs:

  For Canakinumab (Ilaris):
  www.ema.europa.eu/humandocs/Humans/EPAR/il...

  For Rilonacept (Arcalyst):
  www.ema.europa.eu/humandocs/Humans/EPAR/ar...

  Access to these treatments will not immediately occur in each of the European Member States - this can take time. Best consult with your doctor or specialist.

  In the USA the FDA provide information here:

  For Canakinumab (Ilaris):
  www.accessdata.fda.gov/scripts/cder/drugsa...

  For Rilonacept (Arcalyst):
  www.accessdata.fda.gov/scripts/cder/drugsa...

  Finally just a word to say that the aim of this post is simply to provide information. The decision to take any of these treatments should be done in consultation with a medical professional.
  One reply to the topic — see all replies

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    rdc-team published over 2 years ago | Originally written in English

    Further information on Canakinumab (Ilaris®) in French published by the French Regulatory Authority - Haute Autorité de Santé is available at the following link:

    bit.ly/ilarisfr

    This document explains the conclusions from the 3 phases of clinical trials, compares the treatment to other available (prescription and off-lable) treatments for each of the CAPS syndromes and details the level of reimbursement in France to patients.