ADVOCACY MEETING at Alexion Pharmaceuticals to Discuss aHUS and Soliris (Aug. 5, 2010)

[Linda Burke]

Alexion Pharmaceuticals, makers of Soliris (eculizumab), asked the Biermanns and I to participate in an advocacy meeting on August 5th, 2010 to discuss issues regarding aHUS patients and families. We very much appreciated their kind invitation to visit Alexion Pharmaceuticals in Conneticut to speak with senior staff. In representing the Foundation for Children with Atypical HUS, we had this unique opportunity to speak about the special challenges facing aHUS patients and parents wishing to obtain Soliris as part of their treatment plan. I attended the meeting on August 5, 2010 and Bill and Cheryl Biermann participated via teleconference. Alexion has prepared a summary of this meeting, and has authorized its post to our interactive website. Summary of Atypical HUS Advocacy Mtg 08 26 10.pdf .

As website administrator for http://www.atypicalhus.org/ , I encourage patients and families to share information, inspiration, and insight as they deal with a diagnosis of atypical HUS and therefore I welcomed the opportunity to meet with Alexion personnel to exchange ideas about patient issues. In representing this online community of The Foundation for Children with Atypical HUS, I presented an overview of key issues and concerns for those aHUS patients/parents interested in using Soliris as part of their treatment plan.

As founders of The Foundation for Children with Atypical HUS the Biermanns have provided critically important services to the aHUS community, utilizing their vision and experience to craft meaningful outreach, information, and support to patients, families, physicians, and researchers who deal with this rare complement disorder. Bill and Cheryl long have been the 'first responders' for newly diagnosed patients and their families, providing a beacon of hope as they light the path for those traveling along the tumultuous journey through aHUS issues. In addition to providing information and support to aHUS patients and their families, the Biermanns have been an integral part of exploring aHUS investigation pathways, providing Foundation grants to researchers, and keeping informed about/connected to researchers persuing innovative treatment options for aHUS patients.

The Alexion staff present at the August 5th meeting exemplified a passion for and commitment to understanding concerns facing aHUS patients, and it was heartening to hear of their genuine interest in researching a possible new treatment option for aHUS patients. The Biermanns and I look forward to continued conversations stemming from our August 5th sessions and certainly pledge to maintain a collaborative view toward offering ideas and input that might help the Alexion team better understand and support aHUS patients and their families, including those who are receiving Soliris as part of a clinical trial or on an off-label basis. Our advocacy meeting at Alexion allowed us to present your concerns and issues regarding Soliris use for both adult and pediatric aHUS patients, and for renal transplant supportive therapy as well as ongoing Soliris treatment with the goal of preventing hemoylsis. Naturally, it was in the best interest of our aHUS community to give Alexion input about the types of issues facing our aHUS online community. How nice to have Alexion take a proactive approach as they continue to research Soliris and prepare for the possibility of building information/education programs for both patients and physicians.

Future information pertaining to Soliris will be forthcoming, but the same federal regulations put in place to protect patients have the downside that regulatory mandates allow only certain information to be released at strictly regulated junctures in time. On behalf of those struggling with a diagnosis of aHUS, we extend our gratitude and deep appreciation to all whose endeavors move patients and their families forward to a brighter future.

[Dana M Simone]

Wonderful news....we have a family meeting with Jack's Nephrology, CAPD and Transplant team members next week, and I'll have the Alexion / Soliris summary in hand. We have been discussing Soliris for some time and remain hopeful that Jack will have a transplant before turning 18 (next July).

[Deborah Deffenbaugh]

Thanks for your hard work and efforts on behalf of A-HUS. You and the Biermanns are such a pivotal source of information for all of us struggling to make sense of treatment options, etc. and it so helps to know someone is advocating for us--- please know you are truly appreciated. I sometimes feel I get more information from this site than I do the medical community.

[Cheryl Biermann]

Thank you for putting together this summary, Linda. The hope we all have for the future of our loved ones was definitely increased after speaking with and hearing the commitment of the Alexion team.

[Kerri Grey]

Thank you so much to Linda and the Biermanns for advocating on behalf of us all!!!

[Linda Burke]

Alexion certainly is to be much commended for their outreach to patients - hopefully this will be a promising treatment that is a life-changing medicine for many who are battling an ultra-rare disease.
The Biermanns have paved the path of education and support for aHUS patients and their families - their years of hard work on the aHUS battlefront is yielding many such successful advances that continue to unfold for us all.

[Colette Ann Frysz]

Linda,
Kudos to you and the Biermans for being able to bring forth information to the resource of something that could help so many. Kudos to Alexion for even asking.

[Jessica Olivia Frysz]

Linda that is such an honor you received! I'm glad to hear that you are participating and speaking on behalf of the parents and patients affiliated with the disease and maybe that will get some more ideas into their heads that this drug can do wonders for patients with aHUS and hopefully get the drug approved for both patients with the disease already and also for those about to receive transplants, that's GREAT, goodluck!