2019
DOI: 10.1212/wnl.0000000000008207
|View full text |Cite
|
Sign up to set email alerts
|

Combination therapy with nusinersen and AVXS-101 in SMA type 1

Help me understand this report

Search citation statements

Order By: Relevance

Paper Sections

Select...
2
1
1
1

Citation Types

0
27
0

Year Published

2020
2020
2023
2023

Publication Types

Select...
9

Relationship

0
9

Authors

Journals

citations
Cited by 37 publications
(28 citation statements)
references
References 7 publications
0
27
0
Order By: Relevance
“…Clinical trials data on combination therapy are lacking. Lee et al 14 first reported 2 type 1 SMA patients who were treated with nusinersen followed by onasemnogene. In this report, we describe our experience in using combination molecular therapies to treat patients with type 1 SMA in the “real‐world” clinical setting.…”
Section: Discussionmentioning
confidence: 99%
“…Clinical trials data on combination therapy are lacking. Lee et al 14 first reported 2 type 1 SMA patients who were treated with nusinersen followed by onasemnogene. In this report, we describe our experience in using combination molecular therapies to treat patients with type 1 SMA in the “real‐world” clinical setting.…”
Section: Discussionmentioning
confidence: 99%
“…Excitingly, a combined approach using SMN-dependent ASO-inducing SMN2 exon inclusion and SMN-independent myostatin inhibition have shown a favorable result in an SMA animal model [120]. Combined treatment with Zolgensma and nusinersen has recently been investigated in a small group of patients, although the long-term benefit is still unclear [121]. Zolgensma and nusinersen have different mechanisms of action, so the drug-to-drug interaction is less likely.…”
Section: Combination Therapy For Smamentioning
confidence: 99%
“…Head to head studies comparing the amount of SMN protein expression or clinical effect size are not available. The combination of both approaches has also not been studied systematically and warrants further investigation [24]. However, from a theoretical point of view one would not expect the combinational effect to be equal to the sum of their single effects due to the common downstream pathway and mode of action, unless the biodistribution of the different therapeutic compounds was substantially different.…”
Section: Strength Of Consensus: Strong Consensus (100%)mentioning
confidence: 99%