Astellas’ gene therapy for rare disease X-linked myotubular myopathy (XLMTM) has been hampered by safety concerns, so its decision to license an alternative from start-up
The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare genetic disease characterised by fragile skin that can split
In the latest instalment of the pharmaphorum podcast, editor-in-chief Jonah Comstock connects with Kylie O’Keefe to talk about gene therapy in rare diseases.