fly again. This was followed by production of clinical grade alpha-glucosidase in Chinese hamster ovary (CHO) cells and in the milk of transgenic rabbits. This work eventually culminated in the start of clinical trials with the first clinical trial including 4 babies receiving enzyme from rabbit milk at Erasmus MC Sophia Children's Hospital and 3 babies receiving enzyme grown in CHO cells at Duke University in 1999. The currently approved Myozyme is manufactured by Genzyme Corp. in Cambridge, Massachusetts. Its development was a complex process. Genzyme first partnered with Pharming Group NV who had managed to produce acid alpha-glucosidase from the milk of transgenic rabbits. They also partnered with a second group based at Duke University using Chinese hamster ovary cells. In 2001, Genzyme acquired Novazyme which was also working on this enzyme. Genzyme also had its own product (Myozyme) grown in CHO cells under development. In November 2001, Genzyme chief executive Henri Termeer organised a systematic comparison of the various potential drugs in a mouse model of Pompe disease. It was found that the Duke enzyme was the most efficacious, followed by Myozyme. However, due to easier manufacture of Myozyme, work on the other products was discontinued. Funding for research in this field was in part provided by the Muscular Dystrophy Association and the Acid Maltase Deficiency Association in the US, and by the Association of Glycogen Storage Disorders in the UK, as well as the International Pompe Association. John Crowley became involved in the fund-raising efforts in 1998 after two of his children were diagnosed with Pompe. He joined the company Novazyme in 1999, which was working on enzyme replacement treatment for Pompe. Novazyme was sold to Genzyme in 2001 for over US$100 million. The 2010 film Extraordinary Measures is based on Crowley's search for a cure. As of 2019, many biomedical companies are developing Gene therapy in hopes of helping the body create alpha-glucosidase on its own. In 2021, in utero infusions were provided to the fetus of an Ottawa, Ontario, mother who had had two previous children with Pompe disease. The medical team was a collaboration between an Ottawa group and a group at the University of California, San Francisco. The child, born in June 2021, is thriving as of November 2022. References External links GeneReview/NIH/UW entry on Glycogen Storage Disease Type II (Pompe Disease) Understanding Pompe Disease - US National Institute of Arthritis and Musculoskeletal