... I'm not sure whether the $150 is for shipping the biopsy, or the cost of testing once it gets to Children's. When I had my biopsy shipped for testing, the...
I came across the following trial of the steroid Deflazacort which is being investigated in Germany in patients with dysferlin deficiency. Deflazacort is also...
Hi everyone, May I ask all of you what you think of this clinical trial and the use of steroid? And If you are familiar with "deflazacort"= Calcort* ? I would...
Does anyone have the stats concerning the number of folks in the US with dysferlinopathy ? Just curious . I emailed the MDA and they actually referred me to...
... There isn't much in the way of reliable data (at least in the US) partly because there isn't any central place to collect it. Statistics collected on the...
Thanks for the reply Brad. It is very much appreciated. Maybe I'm naive , but I was very surprised the MDA didn't have any answers. Thanks again for your time....
Thanks for the reply Brad. It is very much appreciated. Maybe I'm naive , but I was very surprised the MDA didn't have any answers. Thanks again for your time....
Would it be appropriate to question the Neurological Society, or whatever it's called? Or, maybe better - to submit a questionaire for sending to their members...
What do I think about the clinical trial? There have been some studies testing Deflazacort in Duchenne MD and comparing it to Prednisone in particular. The...
Hi, Honestly, I don't know if real statistics incidence or prevalence focused on dyferlinopathies were studied. It may be a research subject still going on,...
It's true that there aren't good epidemiological studies for many of the rarer subtypes of muscular dystrophy. Dr. Jerry Mendell at Ohio State University...
Since there have been a few questions about appropriate physical therapy from time to time, I thought I'd let you know that there will be a chat on this...
I would like to further my education in this matter. I am an avid reader and would like suggestions on what to purchase. Technical manuals will do little for...
There is an excellent book by Alan Emery called "Muscular Dystrophy: The Facts." It is very comprehensive, but it is aimed at the lay person and includes...
... Daniel: Exactly what sort of information were you looking for? Muscular dystrophy in general or dysferlinopathy in particular? Were you more interested in...
Thanks again Sharon. You are a great help as always. Daniel, I would like something regarding dysferlinopathy if possible, but I would settle for anything...
... For the time being, dysferlinopathy (or even LGMD) is probably too narrow a topic for somebody to write a book about. There are some review articles in...
Dave, May I ask you what form (Miyoshi distal form / LGMD2 B form) of dysferlinopathies you have? Concerning books references, I don't think being helpful...
Hello all, :) Hope you're doing well. Someone asked me about the decrypton, so I thought you might be interested too. This is my 2 cents about it: In France...
There are 8 presentations and one discussion workshop on dysferlin in the program for the upcoming Internatioal Congress on Neuromuscular Diseases in July...
I've decided to activate the "moderater" feature on posting messages. Most people recieve all the postings as e-mails (you can change this under the options...
Thanks, Brad. I think we do need you stepping in. Not that I get all that much mail from the group, but it's a bother when it's a repeat or a spam attack. ...
... Daniel: Thanks for the mention, Brad! While I won't be able to attend the Congress myself, several people from my lab will be paying close attention to the...
Dear members, Since the dysferlin gene is known, why antisense/sense paradigm has not been used more intensively in clinical research? Ce paradigm is based on...
... Dear Sophareth, Thanks for answering and furthering my questions in a so unexpected way, though. I was thinking that antisense/ sense RNA paradigm's...
