Upper Limb Changes in DMD Patients Amenable to Skipping Exons 44, 45, 51 and 53: A 24-Month Study.
Claudia BrognaMarika PaneGiorgia CorattiAdele D'AmicoElena PegoraroLuca BelloValeria Ada Maria SansoneEmilio AlbamonteSonia MessinaAntonella PiniMaria Grazia D'AngeloClaudio BrunoTiziana MonginiFederica Silvia RicciAngela BerardinelliRoberta BattiniRiccardo MassonEnrico Silvio BertiniLuisa PolitanoEugenio Mercurinull Italian Dmd GroupPublished in: Children (Basel, Switzerland) (2023)
Our results expand the information on upper limb function changes detected by the PUL 2.0 in a relatively large group of DMD patients with distinct exon-skipping classes. This information can be of help when designing clinical trials or in the interpretation of the real world data including non-ambulant patients.