As part of our ongoing work on Lysosomal Acid Lipase Deficiency (LAL D) Respondent Journey, sometimes called Cholesterol ester storage disease (CESD) or (Wolman Disease). Market Vision Research is working with Field Global to conduct research about how LAL D impacts patient’s life and how patient’s feel about the medical treatment for this condition. We are interested in patient’s opinions for “LAL D Respondent Journey” for research purpose only. I would like to reassure you that this study satisfies all requirements for a bona fide market research study; there will be absolutely no sales or promotion activities during or resulting from the discussion.
In appreciation of you referring the patient (in case of a Doctor) or agreeing to participate (in case of a patient), following Incentive would be provided.
Patients would be provided with a token of 140 USD for successful interview through telephone and 240 USD for Facility interview in New York.
Hi ! Im 42 , and have had high alt and ast liver function tests , after many months the gastro sent me for a liver biopsy after getting a second opinion the pathologist said it was LAL D . I now am awaiting a genetic blood test for it . I am very scared and need more information , the gastro called it a "vitamin defiency" which clearly it is not . Any information about you or anyone elses story would be appreciated ! thank
Hi Dorothy, I am 46 yo and was diagnosed with LAL D after two years of being sent to just about every specialist that you can think of. Once I was diagnosed, it was a bittersweet moment. I found out why my enzymes were elevated, but there was no treatment. In 2011, a biopharmaceutical company called Synageva started a clinical trial for patients with LAL D (aka cholesterol ester storage disease and wolman's disease). I was the first patient to be infused with a genetically produced enzyme. I am still on the trial. When I first started the trial, my liver biopsy showed that I had an extremely fatty liver. They did another liver biopsy just over a year ago which showed that my liver was no longer fatty and my cholesterol was back in the normal range. The enzyme replacement therapy clinical trial has shown some impressive results for those who were in the trial. It has saved several babies who were born with the most severe form of the disease. Those babies are living happy normal lives with this treatment.
The medication is introduced intravenously once every other week. It was FDA approved late last year. When you get your blood test results back, I would contact someone from Alexion who purchased Synageva last year. Alexis has a group of nurses who assist patients with getting their health insurance to cover the treatment and attempts to find ways to assist with the co-pay. This team from Alexion is called One Source. If you look up Alexion and find a number, you can have them put you in Contact with One Source. If you do not have any luck, let me know. I will make sure that I put you in contact with the correct person. Please let me know if you have any other questions. I know that it is a scary time right now. Hang in there.
Lakhan sutar
LAL D Respondent Journey
Email invite
May 2015
Email title: LAL D Respondent Journey
Dear Sir/Doctor/Respondent,
As part of our ongoing work on Lysosomal Acid Lipase Deficiency (LAL D) Respondent Journey, sometimes called Cholesterol ester storage disease (CESD) or (Wolman Disease). Market Vision Research is working with Field Global to conduct research about how LAL D impacts patient’s life and how patient’s feel about the medical treatment for this condition. We are interested in patient’s opinions for “LAL D Respondent Journey” for research purpose only. I would like to reassure you that this study satisfies all requirements for a bona fide market research study; there will be absolutely no sales or promotion activities during or resulting from the discussion.
In appreciation of you referring the patient (in case of a Doctor) or agreeing to participate (in case of a patient), following Incentive would be provided.
If you have any questions, please contact us via e-mail at Lakhan.vishnusutar@fieldglobal.in
Thank you,
Lakhan sutar
Project Executive
Primary Research Management
Field Global
May 18, 2015
Dorothy
Hi ! Im 42 , and have had high alt and ast liver function tests , after many months the gastro sent me for a liver biopsy after getting a second opinion the pathologist said it was LAL D . I now am awaiting a genetic blood test for it . I am very scared and need more information , the gastro called it a "vitamin defiency" which clearly it is not . Any information about you or anyone elses story would be appreciated ! thank
Feb 27, 2016
Brett B.
Hi Dorothy, I am 46 yo and was diagnosed with LAL D after two years of being sent to just about every specialist that you can think of. Once I was diagnosed, it was a bittersweet moment. I found out why my enzymes were elevated, but there was no treatment. In 2011, a biopharmaceutical company called Synageva started a clinical trial for patients with LAL D (aka cholesterol ester storage disease and wolman's disease). I was the first patient to be infused with a genetically produced enzyme. I am still on the trial. When I first started the trial, my liver biopsy showed that I had an extremely fatty liver. They did another liver biopsy just over a year ago which showed that my liver was no longer fatty and my cholesterol was back in the normal range. The enzyme replacement therapy clinical trial has shown some impressive results for those who were in the trial. It has saved several babies who were born with the most severe form of the disease. Those babies are living happy normal lives with this treatment.
The medication is introduced intravenously once every other week. It was FDA approved late last year. When you get your blood test results back, I would contact someone from Alexion who purchased Synageva last year. Alexis has a group of nurses who assist patients with getting their health insurance to cover the treatment and attempts to find ways to assist with the co-pay. This team from Alexion is called One Source. If you look up Alexion and find a number, you can have them put you in Contact with One Source. If you do not have any luck, let me know. I will make sure that I put you in contact with the correct person. Please let me know if you have any other questions. I know that it is a scary time right now. Hang in there.
Brett
Feb 27, 2016