The European Medicine Agency recently granted orphan designation* to the virus containing MTM1 for the gene therapy for x-linked myotubular myopathy (XLMTM). To read more about this designation, please go to the Website of the EMA.
*A status assigned to a medicine intended for use against a rare condition. The medicine must fulfil certain criteria for designation as an orphan medicine so that it can benefit from incentives such as protection from competition once on the market.
What is the EMA?
The European Medicines Agency is a decentralised agency of the European Union, located in London. The Agency is responsible for the scientific evaluation of medicines developed by pharmaceutical companies for use in the European Union. It began operating in 1995.