Long-term prognosis of fatty-acid oxidation disorders in adults: Optimism despite the limited effective therapies available.
Alice RouyerCéline TardAnne-Frédérique DesseinMarco SpinazziAnne-Laure Bédat-MilletDalia Dimitri-BoulosAleksandra Nadaj-PaklezaJean-Baptiste ChansonGuillaume NicolasClaire DouillardPascal LaforêtPublished in: European journal of neurology (2023)
A standardized register would complete this cohort description of muscular forms of FAODs with exhaustive data, making it possible to assess the efficacy of therapeutic protocols in real-life conditions and during the long-term follow-up of patients.