The Foundation for Children with Atypical HUS

FALL - New Commencement of Pediatric Clinical Trial for Soliris

As part of Alexion Pharmaceutical's second quarter review, the following information was released this week:

Because we are keenly aware that aHUS frequently affects children, often with devastating consequences, we are working expeditiously to expand our aHUS development program to include pediatric patients. As you may be aware, there is a requirement in Europe to submit a formal Pediatric Investigational Plan defining a sponsor’s clinical development program across all pediatric age groups. Based on the regulatory feedback we have received on this Plan, we are now preparing to commence our pediatric aHUS study this fall [in the United States and other countries].

You'll note that the pediatric clinical trial for Soliris now has a fall timeframe, for the reasons described above. I have no further verifiable information to release at this juncture, but will keep you posted when further information comes my way. You are welcome to email any breaking news (complete with source notations for verification purposes) to me at linda@atypicalhus.org .

Views: 0

Comment

You need to be a member of The Foundation for Children with Atypical HUS to add comments!

Join The Foundation for Children with Atypical HUS

Comment by Cheryl Biermann on July 25, 2010 at 8:53pm
Thank you for the hours of work you do Linda, to get this information to the aHUS family!
Comment by Jessica Olivia Frysz on July 24, 2010 at 12:17pm
Thank you so much for the news :) That's good they are going to work on a trial, because I think this drug will do good for patients with aHUS including myself, who is also looking to receive a kidney transplant :)
Comment by Svetlana Finley on July 24, 2010 at 12:47am
Thanks for the news ;-)

WELCOME - Friends, Family Members, Patients, and Researchers - JOIN US!

The Foundation for Children with Atypical HUS encourages patients and investigators to share information and explore options/resources as we work together to gain insight into this rare complement disorder. By increasing contact opportunities with researchers and medical personnel interested in helping the aHUS community, our stories foster a better understanding of atypical hemolytic uremic syndrome.

Sharing information, inspiration and support for one another, we seek to gather together people and knowledge as we strive to improve the lives of patients and families dealing with a diagnosis of aHUS.


NEW DIAGNOSIS OF aHUS?
Be proactive! Get the medical basics of aHUS, what lab values to monitor, and areas of concern...check out the "aHUS Bootcamp" and "About aHUS" tabs at the top of this page!
If your doctor has never treated a case of aHUS, please print out our 'Doc to Doc Registry' and ask him/her to contact a physician versed in the complexities of aHUS and new options for 2011 genetic testing and treatment.

************************

NOTE: Our 'Send a Message" function on each Member's page allows for private discussion of personal content. As with any social network, be cautious about giving personal contact information (home email info, phone number) until you have an established relationship with another person, organization, or associated website.

 

Did you know...

CFH (Serum Complement Factor H) is a regulatory protein. The secreted protein product of CFH consists of 20 repetitive units named "short consensus repeats" or SCRs (each approximately 60 amino acids). In patients with aHUS the last 5 "pearls" in the twenty pearl strand protein, SCR16 - SCR20, should bind to protect cells but do not- they are defective in one or more of the last 5 SCR locations. If they cannot bind or stick to the kidney to protect that tissue, the platelets clump into clots that affect the glomeruli of the kidney -potentially causing acute renal failure.
  
• • • • • • • • • • • •
  
It is estimated that there are about 300 cases of aHUS in the U.S., and it is most common with young children. The condition is life threatening and either can be chronic or can recur at intervals.
  
more factoids...

Help us fight the battle

Your tax-deductible purchase of one or more bracelets will directly fund research to help end the tragedy and heartbreak that aHUS families live with every day. Each bracelet has an appraised value of $825, but your purchase price is only $295.

  

  
Or you can donate a specific dollar amount-every dollar will help the Foundation for Children with atypical HUS atypicalHUS.50megs.com


Donations may be made via credit card or Paypal. If you prefer, checks made payable to The Foundation for Children with Atypical HUS may be mailed to:
Atypical aHUS Foundation
19 Olde Colony Lane
Cape Elizabeth, ME 04107
For pearl bracelet orders, please allow extra time for processing checks.



Badge

Loading…

© 2012   Created by ALPHA MARKETING.   Powered by

Badges  |  Report an Issue  |  Terms of Service