New results for risdiplam in spinal muscular atrophy

Iannaccone, Susan T.

doi:10.1016/s1474-4422(22)00427-6

Cited by 1 publication

(2 citation statements)

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“…Durations for bridging and switching were adopted as reasonable surrogates for distinguishing these two treatment patterns in the absence of direct knowledge of caregiver/provider intent. Definitions for discontinuation were adopted based on previously published/presented analyses 15,16 . Importantly, the actual time on nusinersen therapy before discontinuation is variable depending on phase of treatment (loading doses or maintenance doses).…”

Section: Resultsmentioning

confidence: 99%

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Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification

Proud,

Mercuri,

Finkel

et al. 2023

Ann Clin Transl Neurol

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We sought to devise a rational, systematic approach for defining/grouping survival motor neuron‐targeted disease‐modifying treatment (DMT) scenarios. The proposed classification is primarily based on a two‐part differentiation: initial DMT, and persistence/discontinuation of subsequent DMT(s). Treatment categories were identified: monotherapy add‐on, transient add‐on, combination with onasemnogene abeparvovec, bridging to onasemnogene abeparvovec, and switching to onasemnogene abeparvovec. We validated this approach by applying the classification to the 443 patients currently in the RESTORE registry and explored the demographics of these different groups of patients. This work forms the basis to explore the safety and efficacy profile of the different combinations of DMT in SMA.

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Section: Resultsmentioning

confidence: 99%

“…Definitions for discontinuation were adopted based on previously published/presented analyses. 15,16 Importantly, the actual time on nusinersen therapy before discontinuation is variable depending on phase of treatment (loading doses or maintenance doses).…”

Section: Resultsmentioning

confidence: 99%