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18.05.2020 back forward

Marketing Authorisation of Gene Therapy

DNA (Source: Qimono / Pixabay)

On 18 May, the European Commission authorises a gene therapy medicine for the treatment of certain forms of spinal muscular atrophy. One in 10,000 children is affected by this genetically caused muscle wasting which, if left untreated, can have a fatal outcome during childhood. The Paul-Ehrlich-Institut supports the development of urgently needed gene therapies through to their marketing authorisation – and thus brings them to patients.

Gene Therapy for Muscular Atrophy