LAL Deficiency (Lysosomal Acid Lipase Disease) & Wolman Disease patient support
It is hard to believe that it has been nearly three years since I started the Sebelipase Alfa clinical trial. Before I agreed to participate in the study, I made several phone calls to many of my physicians and to the National Institute of Health to gather additional information on Enzyme Replacement Therapy. None of them had any concerns and felt that it would be beneficial to my health to participate in the clinical trial. As many of you are participating in the ARISE study, I can clearly remember sitting in the hospital bed getting ready for my very first infusion at Mount Sinai School of Medicine in New York City. The hospital staff made me feel very comfortable. I brought my computer along so I could get some work done during the infusion. The first infusion kept the nurses busy, but I was able to concentrate and get some things accomplished. I have been asked by many people if it feels any different during your infusion. The answer is no. I do not feel any different before, during or after the infusion. I have not had any adverse reactions to the medication.
Eventually, my care was transferred to the University of Minnesota which is a lot closer to home. It has become a routine to go to the U of M to get my infustions every other week. Just as I did in New York, I always bring something along to keep me busy. Recently, there has been data released about this phase of the clinical trial, which will give you a better understanding about how the medication has helped those of us who have been participating in the initial phase of the trial. I can not speak on behalf of the other participants, but my results have been astounding and I do feel better. For those of you who are getting ready to participate in the clinical trial or those of you who have recently started you infusions who have any questions about my experience with the trial, feel free to contact me. I will not provide any medical advice, but will share my experience.
Never Give Up!