Intraputaminal Gene Delivery in Two Patients with Aromatic L-Amino Acid Decarboxylase Deficiency.
Marie-Céline François-HeudeGaetan PoulenEmmanuel Flamand RozeMarie-Ange Nguyen MorelDomitille GrasIsabelle Roch-ToreillesAdeline QuintardGaelle BarouxPierre MeyerPhilippe CoubesChristophe MilesiGilles CambonieJulien BaleineChrystelle SolaBénédicte DelyeEvgenia DimopoulouStéphanie SanchezMathieu GasnierSouad TouatiAlberto ZamoraDaniel PontalNicolas LeboucqVirginie KouyoumdjianAdrien LebasnierSylvia SanquerDenis Mariano-GoulartThomas RoujeauAgathe RoubertiePublished in: Movement disorders clinical practice (2023)
Two patients with a severe form of AADCD had an objective motor and non-motor benefit from eladocagene exuparvovec injection even when treated after the age of 10 years, as in the seminal study.
Keyphrases