This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The objective of the Myozyme Temporary Access Program is to provide adult patients with Pompe disease in the US access to Myozyme produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration (FDA). Myozyme has been used since early 2003 in clinical trials to treat patients with Pompe disease. Market approval in the US for the use of Myozyme to treat Pompe disease was granted on 28 April 2006. Genzyme has scaled up and modified the existing manufacturing process for production of Myozyme at a larger scale, which is currently approved for commercial use outside of the US, in order to provide a continued supply to patients. This scale of material is not yet pproved for commercial use in the US;however it is used in ongoing clinical trials. Myozyme, produced from the scaled-up manufacturing process will be administered for a limited time under this access program until the manufacturing scale is approved by the FDA.
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