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Last Updated: 08/26/2022

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Found 53 publications

Clinical follow-up analysis of nusinersen in the disease-modifying treatment of pediatric spinal muscular atrophy.

Summary: Clinical follow-up analysis of nusinersen in the disease-modifying treatment of pediatric spinal muscular atrophy.

Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular Atrophy.

Summary: Safety, tolerability, and efficacy of a widely available nusinersen program for Polish children with Spinal Muscular Atrophy.

Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

Summary: Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial.

An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

Summary: An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.

Summary: Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.

Respiratory outcome of spinal muscular atrophy type 1 patients treated with nusinersen.

Summary: Respiratory outcome of spinal muscular atrophy type 1 patients treated with nusinersen.

Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland.

Summary: Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland.

A Case Report of Systemic Intoxication Following Onabotulinum Toxin A Injections Into the Salivary Glands in a Patient With Spinal Muscular Atrophy Type 1.

Summary: A Case Report of Systemic Intoxication Following Onabotulinum Toxin A Injections Into the Salivary Glands in a Patient With Spinal Muscular Atrophy Type 1.

Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.

Summary: Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial.

Nusinersen: A Review in 5q Spinal Muscular Atrophy.

Summary: Nusinersen: A Review in 5q Spinal Muscular Atrophy.

Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.

Summary: Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study.

Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.

Summary: Nusinersen for Spinal Muscular Atrophy in the United States: Findings From a Retrospective Claims Database Analysis.
Showing 1-12 of 53

Last Updated: 08/26/2022