My mom was diagnosed with the disease within the past year and so we are intimately familiar with its symptoms and the impact it has on the lives of those affected. We are appreciative for the work that Dave Hill has done in maintaining a database of others who have been diagnosed with SS.
I was hoping to get more information so that we could further our research as we deliberate on a treatment plan that incorporates the drug. I am writing from the U.S. where Deferiprone is not yet approved by the FDA and thus not readily available which also presents some challenges that we are looking to overcome.
I look forward to hearing back from you and hope you are doing well in the meantime,
Last edited by Mod-S4; 09-28-2011 at 07:38 PM.
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Your best bet is to get in touch with Dr. Michael Levy at Johns Hopkins in Baltimore. He is the only physician I was ever able to find that is knowledgeable about this disease. He is running a trial/ study about SS and Ferriprox. He was able to gain FDA approval for my father to try it. We did see some success in the early days--but my Father's condition was really advanced by the time it was properly diagnosed. The sooner your Mom can try, the better.
We are in touch with Dr. Levy We are now just debating whether or not to pursue treatment with Deferiprone (as the next trial is not expected to begin for several months) and looking to speak with people who have used it to understand their experiences, the risks, and how they were able to procure the drug.
Sounds like you saw some improvement during the trial, albeit the trial was only for a short time? I would be curious to learn more if you are available to speak or via email?
Also, if there is anyone else out there who has experience with Deferiprone I would be curious to hear about your experience as we embark on a treatment using the drug.
Last edited by moderator2; 07-26-2011 at 10:10 AM.
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Well, I can tell you that my father didn't really experience any bad / harmful side effects. The only side effect we saw was the expected change in urine color. With his first 3 month trial we saw improvement in his coordination and a decrease in his dementia. It's hard to be any more specific than that because the changes were subtle. Then, if I recall correctly it was like 2 years later when he started it again. By that point, he was so far disabled that we couldn't really assess any improvement. We took him off the drug to ensure that the trial was not skewed by him. He passed away in January. The last year he was in a nursing home (at 64) and the year before that he required round the clock care at home. This disease is tragic.
I am very sorry for you loss. It is indeed a very tragic disease and heartbreaking to see a loved one suffer through it. I appreciate you taking the time to respond and to discuss your experience. From our limited research and our discussions with Dr Levy it sounds as though Deferiprone has had some positive impacts. Unfortunately the next phase of the study is not likely to beginfor some time so we do not really have a way of obtaining the drug unless we go overseas which are considering. In the meantime, trying to assess others' experiences to better understand the drug and its side effects. So thank you once again for your response and your story.
Unfortunately I have not heard from the others on this board but would be helpful if anyone else who has experience with superficial siderosis and/or Deferiprone/Ferriprox could respond as well.