Titre : | Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment |
Revue : | Journal of Neuromuscular Diseases |
Auteurs : | McGrattan KE ; Shell RD ; Hurst-Davis R ; Young SD ; O'Brien E ; Lavrov A ; Wallach S ; LaMarca N ; Reyna SP ; Darras BT |
Type de document : | Article |
Editeur : | Netherlands, 17/04/2023 |
Note générale : |
Erratum to: Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment
On page 536, for Figure 2, the figure legend was incorrect from line 5 onwards. The inaccurate text is: At baseline, communication was assessed for 20 native-English speaking patients in STR1VE-US who had a CHOP INTEND score of 60 or greater; communication was assessed before end of study for four patients in START and 16 in STR1VE-US. This text should be replaced with: Communication was assessed for patients from native English-speaking families in START and STR1VE-US. In addition, communication was only assessed for patients in START who achieved a CHOP INTEND score≥60. At baseline, communication was assessed for 20 patients in STR1VE-US; communication was assessed before end of study for four patients in START and 16 in STR1VE-US. The Figure 2 including the complete and correct legend is included |
Langues: | Anglais |
Mots-clés : | alimentation-nutrition ; amyotrophie spinale ; amyotrophie spinale proximale liée à SMN1 ; amyotrophie spinale proximale type 1 ; atteinte bulbaire ; déglutition ; efficacité thérapeutique ; essai clinique ; onasemnogene abeparvovec-xioi ; thérapie génique |
Pubmed / DOI : | Pubmed : 37092232 / DOI : 10.3233/JND-221531 / Pubmed : 37661906 / DOI : 10.3233/JND-239002 |
N° Profil MNM : | 2023051 |
En ligne : | http://www.ncbi.nlm.nih.gov/pubmed/37092232 |
Voir aussi : |