O USO DO NUSINERSEN NO TRATAMENTO DA ATROFIA MUSCULAR ESPINHAL: REVISÃO DE LITERATURA
Elzanice Monteiro DOMINGOS+1 more
openalex +2 more sources
Compilation of online resources of relevance to 'Spinraza and advanced therapies:a stakeholder special' issue of Gene Therapy [PDF]
Prakash, V, Yáñez-Muñoz, R J
core +1 more source
Effect of nusinersen on motor, respiratory and bulbar function in early-onset spinal muscular atrophy [PDF]
Baumann, Matthias+39 more
core +1 more source
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam [PDF]
Risdiplam is a once-daily oral, survival of motor neuron 2 (SMN2) splicing modifier approved for the treatment of spinal muscular atrophy (SMA). JEWELFISH (NCT03032172) investigated the safety, tolerability, pharmacokinetics (PK), and PK/pharmacodynamic (
Bruno, Claudio+15 more
core
Post-Marketing Safety of Spinal Muscular Atrophy Therapies: Analysis of Spontaneous Adverse Drug Reactions from EudraVigilance. [PDF]
Belančić A+4 more
europepmc +1 more source
Enhancing the effects of nusinersen with cybernic treatment using Hybrid Assistive Limb (HAL) in spinal muscular atrophy: a real-world case series and exploratory cohort analysis. [PDF]
Nakajima T+8 more
europepmc +1 more source