Diese Resultate dürften die letzten Kritiker zum schweigen bringen. Biogen publizierte ihre neusten Forschungsergebnisse auf der letzten Muscular Konferenz in Virginia. Kinder, Teenager und heranwachsenden Jugendliche mit SMA Typ II und III zeigen eine deutliche Verbesserung ihrer motorischen Funktionen.
These results reinforce SPINRAZA’s effectiveness as the first and only approved treatment for SMA and demonstrate once again the benefit it can bring to individuals with SMA, including infants, teens and young adults“
In the NURTURE study, SPINRAZA was administered to infants six weeks old or younger (n=25), who were in the pre-symptomatic stage, genetically-diagnosed with SMA and had two or three copies of the SMN2 gene (n=15 for two copies (most likely to develop Type 1 SMA); n=10 for three copies (most likely to develop Type 2 SMA)). At the time of this interim analysis, infants had been followed for up to 25.6 months – well beyond the typical timeframe when most infants with Type 1 SMA would have required permanent ventilation or died. The interim analysis, titled, “Nusinersen in Infants Who Initiate Treatment in a Pre-Symptomatic State of SMA: Interim Efficacy and Safety Results from the Phase 2 NURTURE Study,” showed that all infants were alive and none required tracheostomy or permanent ventilation.