第二種檢驗法:酵素活性偵測技術 (Techniques on the Detections of Enzyme Activities)
其測定原理主要是利用已知濃度並且標記有放射線同位素、化學螢光物質、或其他化學呈色物質的基質(substrate)，在酵素的催化作用下，於單位時間內，偵測附著有標記之產物產生的速率。所測得帶有標記產物產生的速率則與參與催化作用之酵素活性呈正比的關係。
Our conference ended yesterday afternoon with great potential for the future for
all MPS I II and III. We were expecting to develop a strategy for Sanfilippo after our meeting and to our great surprise and delight our two major targets are also potential treatments for MPS I and II.
Many great minds came together and discussed their strategies. They all worked with a spirit of collaboration and brought new ideas to each other.
We are very excited at the two possibilities and will be putting together budgets over the next two weeks and begin work immediately afterward.
The most promising development was a small molecule that changes the structure of
heparan sulfate such that the deficient enzyme is not needed to breakdown the heparan
chain. This is a major breakthrough and can be in trials in 2011 (sooner I think if we all pull together on this, MPS I, II and III). Think of the possibilities for MPS I and II even with enzyme replacement, it would be a disease altering medication. It will be a daily oral
medication and it crosses the Blood brain barrier which is needed for this to work.
The second is a promising treatment that is shown to reduce heparan sulfate accumulation in the brain and body. Good results in mice and could slow down all these
diseases significantly.
There is much work to be done and we all need to pull together on this. I would like to see this out sooner that they anticipate and it can be done if we push it through and fund as much of the preliminary work as we can.
If anyone wants to know more please call me 518 879 6571 or Misty 205 238 1151.
This is HUGE for all of us. We need to come together as a community and push these people to translate these to therapies for our children...all our children. We, parents are the only ones that can do it, please consider supporting us. Call us anytime.
Kathy and MIsty
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