Tableau I
Essais cliniques avec traitement par Spinraza® (Nusinersen) (adapté de Claudia A. Chiriboga [13]). AS: amyotrophie spinale; N: nombre des patients; NL: normal; exam neuro: examen neurologique; resp.: ventilation respiratoire permanente, trachéostomie ou ≥ 16 h de ventilation; HINE2: The Hammersmith Infant Neurological Examination; CHOP INTEND: The Children’s Hospital of Philadelphia Infant test for Neuromuscular Disorders; HFMSE: Hammersmith Functional Motor Scale Expanded; 6MWT: test de marche de 6 min; RULM: Revised Upper Limb Module; CMAP: Compound Muscle Action Potential.
| CS3A | Endear (CS3B) | CS2/CS12 | Cherish CS4 | Nurture | |
|---|---|---|---|---|---|
| Sponsor | Ionis | Biogen | Ionis | Biogen | Biogen |
| Design de l’étude | En ouvert | Contrôlé | En ouvert | Control vs procédure placebo | Contrôlé ou en ouvert |
| N | 20 | 123 | 28-58 | 126 | 17 |
| Critères d’inclusion | <7 mois | <7 mois | 2-15 ans | 2-12 ans | Présymptomatique |
| SMA1 | SMN2=2 | SMA 2,3 | SMA2 | Exam neuro NL | |
| SMA1 | CMAP1 mV | ||||
| Evaluations | Survie/resp. | Survie/resp. | HFMSE | HFMSE | Survie/resp. |
| HINE2 | HINE2 | 6MWT | RULM | HINE2 | |
| CHOP INTEND | CHOP INTEND | RULM | CMAP | ||
| CMAP | CMAP | ||||
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