Multidisciplinary team meetings in treatment of spinal muscular atrophy adult patients: a real-life observatory for innovative treatments.
Emmanuelle Salort-CampanaGuilhem SoléArmelle MagotCéline TardJean-Baptiste NouryAnthony BehinElisa De La CruzFrançois BoyerClaire LefeuvreMarion MasingueLouise DebergéArmelle FinetMélanie BrisonMarco SpinazziAntoine PegatSabrina SacconiEdoardo MalfattiAriane ChoumertRémi BellanceAnne-Laure Bedat-MilletLéonard FeassonCarole VuillerotAgnès Jacquin-PiquesMaud MichaudYann PereonTanya StojkovicPascal LaforêtShahram AttarianPascal CintasPublished in: Orphanet journal of rare diseases (2024)
In the context of costly treatments with few controlled studies in adults with SMA, in whom assessment of efficacy can be complex, SMDTs are 'real-world observatories' of great interest to establish national recommendations about indications of treatment and follow-up.