Orphan designation: Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s Treatment of chronic inflammatory demyelinating polyneuropathy, 22/05/2023 Positive

Submitted by Anonymous (not verified) on 24 January 2024 - 12:11

Orphan designation: Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s Treatment of chronic inflammatory demyelinating polyneuropathy, 22/05/2023 Positive

Orphan designation: Adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene Treatment of Dravet syndrome, 22/05/2023 Positive

Submitted by Anonymous (not verified) on 24 January 2024 - 12:06

Orphan designation: Adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene Treatment of Dravet syndrome, 22/05/2023 Positive

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