Nusinersen treatment of older children and adults with spinal muscular atrophy

Nusinersen efficacy data for 24‐month in type 2 and 3 spinal muscular atrophy

“…Our results confirm the previously reported increase in motor function after 12 months of nusinersen treatment 5,7,9,10,20 but also show that further improvement, even if smaller, can also be observed in the second year. The increase was more obvious on the HFMSE, with changes reaching significance already after 12 months in type 2.…”

“…In conclusion, our study confirms that an improvement in nusinersen-treated patients can also be observed in the second year, as suggested by the follow-up of clinical trials 5 or in smaller cohorts of treated patients. 9,10 Our study also strongly suggests that comparison with available natural history data in untreated patients can therefore help to set up correct expectations and to better identify responders in patients of different age and functional level. As a number of patients are considering switching to a new drug after 1 year of treatment, our longitudinal 24-month data using monotherapy with nusinersen will help to better understand possible differences with the changes observed following the introduction of a new or concomitant drug.…”

“…[5][6][7][8] Less has been reported in type 2 and 3 patients. 5,9,10 The aim of this study was to report on real world data in type 2 and 3 SMA patients, including children and adults, treated for at least 2 years with nusinersen and to compare them to available natural history data.…”

Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis

“…Most of the early real-world data have focused on type 1 infants enrolled in early access programs, [8][9][10] but in the last few years several studies have reported additional data in older children [11][12][13][14] and adults [15][16][17][18][19][20][21][22][23][24][25][26][27][28][29], covering the whole spectrum of SMA, from young infants with the most severe forms [30] to adults with a milder phenotype. The real-world data have expanded our knowledge on safety and efficacy of the drug in a much larger population of SMA patients than those reported in the pivotal studies.…”

Spinal muscular atrophy: from rags to riches

“…There is a rapidly increasing number reporting efficacy of the drug in a real world setting. While most of the early real-world data focused on type 1 infants enrolled in early access programs, [37][38][39] there has been a rapidly increase in the number of patients reporting real world data in adults [40][41][42][43][44][45][46][47][48][49][50][51][52][53] and older children [54][55][56][57][58] covering the whole spectrum of SMA, from young infants with the severe neonatal onset forms to adults with milder phenotypes. The real-world data have expanded our knowledge on safety and efficacy of the drug in a much larger population of SMA patients than those reported in the pivotal studies.…”