In order to treat an underserved population of patients with a rare condition whose muscles gradually become weaker until they can no longer walk, a team of researchers across Japan have completed a clinical study to confirm the safety of long-term administration of a therapeutic drug.
Aceneuramic acid is the first approved drug for GNE myopathy treatment retrieved 20 August 2024 from https://medicalxpress.com/news/2024-08-aceneuramic-acid-drug-gne-myopathy.html
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