Old 02-28-2010, 03:53 PM #1 (permalink)
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Default No Compassionage Use of KNS-760704

I wrote to Mr. Petzinger about getting compassionate use for this drug nd this was his response to me. Time is running out for my husband and other ALS patients.

What else can be done? Any ideas?

Dear Aleta,

Please call me Tom.

I want to thank you for your note. We receive many communications from patients and families, as you clearly recognize, but some, such as yours, stand out as real statements from the heart. Itís a reminderóas if we needed oneóabout why we set up our company in the first place. Our sole focus is advancing a meaningful treatment for ALS.

Indeed, thatís the very reason why I have to disappoint your request, much as I might wish it were otherwise.

I donít know the particulars of Charlieís case and, as a layman, Iím certainly unqualified to evaluate it. But as a general matter, getting a drug approved in this disease, especially after so many failures, requires rigorous adherence to protocol requirements, including enrollment criteria. Meeting the goal also means limiting exposure of the drug to people enrolled in well-controlled studies reviewed and approved by regulatory bodies in the U.S. and Europe. To put it simply, we have to conduct our studies by the book if weíre to have the best possible chance of success against this disease.

Finally, we havenít established in a large population that the drug is safe for ALS patients. Weíre especially mindful that in some recent large clinical trials, drug candidate actually worsened the conditions of ALS patients, despite showing earlier promise.

I admit this reasoning may ring hollow to someone living with the disease now, but itís motivated by a commitment to advancing a drug that ultimately may serve the entire community of people with ALS, long into the future.

Thank you again for writing. Best wishes to you and Charlie.

Sincerely,

Tom Petzinger
--------------------------------------------------------

Tom Petzinger
EVP, Business Development and Public Affairs
Knopp Neurosciences Inc.
2100 Wharton Street - Suite 615
Pittsburgh PA USA 15203
+ 412.488.1776
Knopp Neurosciences
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Old 03-01-2010, 09:13 AM #2 (permalink)
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Default Re: No Compassionage Use of KNS-760704

sorry for your disappointment.....and thanks for sharing this with us.
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Old 03-01-2010, 08:21 PM #3 (permalink)
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Default Re: No Compassionage Use of KNS-760704

Aleta, I am so proud of your courage to write the letter and soul sorry that potential medications are withheld from PALS. I feel your heart pain. This is just another reason why I feel sooooo outraged. hugs to you.
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Old 03-02-2010, 12:18 AM #4 (permalink)
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Default Re: No Compassionage Use of KNS-760704

uhg. This is so frustrating! I'm so sorry...
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Old 03-02-2010, 12:33 PM #5 (permalink)
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Default Re: No Compassionage Use of KNS-760704

Thanks all-

I'm not giving up. I think if we can get enough PALs together and request compassionate use, they may prevail.

Any other thoughts?

Aleta
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