Monday, 6 September 2010

Progress - slow and painful

On 2 August BioMarin announced that they were stopping the development of their Utrophin upregulation drug.

Utrophin, from what I can determine, is produced by the body when an infant and performs a similar role to that of dystrophin. As the child grows older the body then switches to producing the more effective dystrophin.

The aim behind this research was to get the body to start producing Utrophin in sufficient quantities to compensate, to some degree, from the lack of Dystrophin produced by the boys.

BioMarin has not stopped all their work - merely this particular process - and so whilst it is not the end of the line it is still a bit disappointing and another reminder of how slow and painful progress can be.

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