The Lancet—July 29, 2000
Recombinant human alpha-glucosidase from rabbit milk in Pompe patients
Lancet 2000; 356: 397-398
Hannerieke Van den Hout, Arnold J J Reuser, Arnold G Vulto, M Christa B Loonen, Adri Cromme-Dijkhuis, Ans T Van der Ploeg
Following is a short summary of the information published:
The results of the effects of the enzymeA protein that helps the body carry out chemical reactions. More, human acid alpha-glucosidase, on four infantile Pompe patients was described by Ans Van der Ploeg, M.D, Ph.D., et al, in this issue of The Lancet Interactive. It documented the successful treatment of 4 babies with the infantile form of Pompe’s disease who were treated with recombinant human alpha-glucosidase from the milk of transgenic rabbits. The results showed that there was uptake of the enzymeA protein that helps the body carry out chemical reactions. More in skeletal muscleMuscles that control movement and are commonly affected in Pompe disease. More and the muscle function was stimulated. The treatment reduced heart size, improved heart function and condition, and seemed to be responsible for the prolonged lives of the four babies in the clinical trialA research study that tests new treatments or approaches in people. More.
Pompe’s disease is a fatal muscle disease caused by the lack or deficiency of the lysosomal enzymeA protein that helps the body carry out chemical reactions. More acid alpha-glucosidase (acid maltase). The infantile form of the disease is usually fatal within the first year of life. All four babies in the trial passed this critical one year juncture.
The report covered the first 36 weeks of the infantile clinical trialA research study that tests new treatments or approaches in people. More which began in January 1999, and is still ongoing in the Netherlands at Sophia Children’s Hospital, an affiliate of Erasmus University, Rotterdam.