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  1. Inotersen for treating hereditary transthyretin amyloidosis (HST9)

    Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

  2. Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

    Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children

  3. Alpha Stim AID (MT477)

    In development [GID-MT542] Expected publication date: 08 March 2021

  4. MT582 AnaConDa

    In development [GID-MT556] Expected publication date: 23 February 2022