Comments on: The Road to Building a New Orphan Drug – Am I Mad? http://addiandcassi.com/the-road-to-building-a-new-orphan-drug-am-i-mad/ Treatments & Cures for Niemann Pick Type C - Childhood Alzheimer's Mon, 06 Feb 2012 20:01:32 +0000 hourly 1 http://wordpress.org/?v=3.3 By: Khalid shaikh http://addiandcassi.com/the-road-to-building-a-new-orphan-drug-am-i-mad/comment-page-1/#comment-2550 Khalid shaikh Wed, 14 Jul 2010 07:21:12 +0000 http://addiandcassi.com/wordpress/?p=2518#comment-2550 Hi, Myself Khalid from India, recently it was identified that my son age 3 is NPC positive. I am very upset and looking for any medicine and supports from you. At present my son is 100% perfect and doing good, with just mild speel enlargment. You may have question why did i go for medical test for NPC for my child since there are no symptoms and answer for that was i had already lost two child due to rare disease NPC??? which was not sure at that time. Hence this time i am really worried. Please help me my identifying medicine /supportive medicine. Below is the gene study data;p.Tyr871SerfsX64 [ c.2612delA] ( this is a new frame shift mutation on Exon 18 ) p. Ile1061Thr [ c.3182 T>C ] Regards Khalid 9109823436565 India,Maharashtra,Pune Hi,
Myself Khalid from India, recently it was identified that my son age 3 is NPC positive. I am very upset and looking for any medicine and supports from you. At present my son is 100% perfect and doing good, with just mild speel enlargment. You may have question why did i go for medical test for NPC for my child since there are no symptoms and answer for that was i had already lost two child due to rare disease NPC??? which was not sure at that time. Hence this time i am really worried. Please help me my identifying medicine /supportive medicine.
Below is the gene study data;p.Tyr871SerfsX64 [ c.2612delA] ( this is a new frame shift mutation on Exon 18 ) p. Ile1061Thr [ c.3182 T>C ]

Regards
Khalid
9109823436565
India,Maharashtra,Pune

]]> By: Chris Hempel http://addiandcassi.com/the-road-to-building-a-new-orphan-drug-am-i-mad/comment-page-1/#comment-2547 Chris Hempel Sat, 10 Jul 2010 04:56:31 +0000 http://addiandcassi.com/wordpress/?p=2518#comment-2547 Hi Lititia: Thank you for your comment. We use Hydroxy Propel Beta Cyclodextrin (HPBCD) which has a very good safety profile and in endotoxin controlled. We use Trappsol brand from CTD in High Springs, Florida, USA. Johnson and Johnson has also done millions of dollars of safety studies on HPBCD -- it's part of one of their drugs called Sporanox. If you have any details on HPBCD being unsafe or toxic, please let me know. Best, Chris Hi Lititia:

Thank you for your comment. We use Hydroxy Propel Beta Cyclodextrin (HPBCD) which has a very good safety profile and in endotoxin controlled. We use Trappsol brand from CTD in High Springs, Florida, USA.

Johnson and Johnson has also done millions of dollars of safety studies on HPBCD — it’s part of one of their drugs called Sporanox.

If you have any details on HPBCD being unsafe or toxic, please let me know.

Best,
Chris

]]> By: Lititia http://addiandcassi.com/the-road-to-building-a-new-orphan-drug-am-i-mad/comment-page-1/#comment-2539 Lititia Mon, 21 Jun 2010 13:33:55 +0000 http://addiandcassi.com/wordpress/?p=2518#comment-2539 Mom, I appreciate and commend your servitude. However please make certain the Cyclodextrins your children are using are NOT derivatives from Lipophilic Cyclodextrin. Lipophilic Cyclodextrin derivatives have shown via laboratory experiments to be toxic. God Bless. Mom, I appreciate and commend your servitude. However please make certain the Cyclodextrins your children are using are NOT derivatives from Lipophilic Cyclodextrin. Lipophilic Cyclodextrin derivatives have shown via laboratory experiments to be toxic. God Bless.

]]> By: Catherine Calhoun http://addiandcassi.com/the-road-to-building-a-new-orphan-drug-am-i-mad/comment-page-1/#comment-2437 Catherine Calhoun Sun, 07 Feb 2010 00:09:11 +0000 http://addiandcassi.com/wordpress/?p=2518#comment-2437 I think your name badge should say "Rock Star" - and you are right! XO, Catherine P.S. I need a "blog for rare" post for a collection of posts over at SNiPs specific for World Rare Disease Day - a mini-rare blogging carnival of sorts planned for the last week of February. Can you send a good link my way? I think your name badge should say “Rock Star” -
and you are right!

XO, Catherine

P.S. I need a “blog for rare” post for a collection of posts over at SNiPs specific for World Rare Disease Day – a mini-rare blogging carnival of sorts planned for the last week of February. Can you send a good link my way?

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