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Retrotope's RT001 Receives EMA's Orphan Drug Designation for Infantile Neuroaxonal Dystrophy (INAD)

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Retrotope's RT001 Receives EMA's Orphan Drug Designation for Infantile Neuroaxonal Dystrophy (INAD)

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  • The EMA’s ODD is based on P-II/III study assessing RT001 in patients with INAD- a neurodegeneration associated with PLA2G6
  • EMA’s ODD is granted to the drug offering treatments for rare- devastating- fatal- and untreated diseases affecting fewer than 200-000 people or >200-000 persons
  • Retrotope’s RT001 is an oral deuterated polyunsaturated fatty acid (D-PUFA) working against PUFA to prevent cell death

Ref: Retrotope | Image: PRNewswire

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