Orphan designation: idebenone Treatment of Friedreich's ataxia, 08/03/2004 Positive

Orphan designation: idebenone Treatment of Duchenne muscular dystrophy, 20/03/2007 Positive

Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Angelman syndrome, 06/01/2021 Positive

Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Phelan-McDermid syndrome, 06/01/2021 Positive

Orphan designation: Cyclo-L-glycyl-L-2-allylproline Treatment of Pitt-Hopkins syndrome, 06/01/2021 Positive

Orphan designation: Metreleptin Treatment of Lawrence syndrome, 17/07/2012 Positive

Orphan designation: Metreleptin Treatment of Berardinelli-Seip syndrome, 17/07/2012 Positive

Orphan designation: Metreleptin Treatment of Barraquer-Simons syndrome, 17/07/2012 Positive

Orphan designation: Metreleptin Treatment of familial partial lipodystrophy, 17/07/2012 Positive

Orphan designation: Humanised IgG4 monoclonal antibody against A proliferation-inducing ligand Treatment of primary IgA nephropathy, 21/06/2022 Positive