My name is nithin. I am Bangalore. I wanted to connect with you on treatment facilities for dmd in Bangalore. Could you let me know your personal email id or phone number So I can connect with you.
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Hello sir. i am from Pathankot, State of Punjab. my two sons are diagnosed with duchchene muscular dystrophy they are 12 years and 10 years old respectively. may god bless your beloved son and whole family.
i think these medicine are used by you, what kind of change you have seen in patient.
the brand name of medicine are Indian or ? These brand are not available in local market I mean not available in Pakistan.
my son now 17y 6 m, Please suggest me can I use these medicine.
When we receive positive news from any one,we feel ourselves energetic
I think Indian Doctors are one step ahead than Pakistan.
Thank you very much for giving me information.
Do you know what kind of medicine are there to make patient strong,
Who is in front line regarding research?Is there any roll of Indian scientist and Doctors regarding DMD?
Accerlon presened a lot of info about the CA trials and other preclinical work related to it. The dosage range is quite high and they admit they are guessing on that. Safety will come first in this phase.
Somebody asked specifically about US trials and the response was "not this year" but that they were working with FDA towards the goal of establishing the right dosage. FDA will be more strict about that and so my *guess* is they won't ask FDA for approval until they have decided a more limited range dosage ranges to study which means they won't say much about that in 'open air" until results are in.
They did mention that they are already planning extension study in CA sites for 2011 after dosage is validated, so that shows some confidence. Also, the overall buzz re: myostatic inhibitors was very high as there are other AAV-based treatments being studied too that work through Follistatin process rather than ACE inhibitor.
Prof. Wilton did comment on multiple skps... in fact there were 2 presentations on that topic using different techniques.
Both groups found that the efficiency of a single cocktail to skip more than 4 exons was compromised. With Western Blot they showed more than 4 would become less and less efficient at protein production.
However, if they used 2 cocktails that each skipped 4 exons the efficiency was good. So at least in theory, the entire 45-55 set could be skipped with 2 cocktails.