PBGM01 – Patient Worthy

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PBGM01 to Treat Infantile GM1 Gangliosidosis Granted Orphan Drug Designation

Post author:Jessica Lynn
Post published:May 25, 2020
Post category:Rare Disease

A gene therapy for the treatment of GM1 gangliosidosis was granted Orphan Drug Designation in April. PBGM01 seeks to treat the most severe form of this condition - infantile. Read…

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