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New gene therapy product Zolgensma to treat spinal muscular atrophy

Posted on: 30 March 2020, source: EMA
The EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma(onasemnogene abeparvovec)to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. There are currently limited treatment options for children with SMAin the EU. Patients also receive physical aids to support muscular functions and help them and their families cope with the symptoms of the disease.