Results 181 to 190 of about 6,101 (249)

The Impact of Nusinersen Treatment on Respiratory Function in Patients with Spinal Muscular Atrophy: A Systematic Review. [PDF]

J Clin Med
Aldukain M, Aldukain A, Hobani A, Barakat A, Alsayyed L, Alomar M, Zain Al-Abeden MS, Alzoum N, Asseri AA.   +8 more
europepmc   +1 more source

Type-1 spinal muscular atrophy cohort before and after disease-modifying therapies. [PDF]

Arq Neuropsiquiatr
Alves BKAMF, Araujo APQC, Santos FND, Ribeiro MG.   +3 more
europepmc   +1 more source

Neurodegeneration Biomarkers in Adult Spinal Muscular Atrophy (SMA) Patients Treated with Nusinersen [PDF]

Pol Andrés‐Benito, Juan F. Vázquez‐Costa, Nancy Carolina Ñungo Garzón, M.J. Colomina, Carla Marco, Laura González, Cristina Terrafeta, Raúl Domínguez, Isidró Ferrer, Mònica Povedano   +9 more
openalex   +1 more source

A real-world pharmacovigilance study of nusinersen based on the FAERS database [PDF]

Weiling Guo, Yi Fang, Aiguang Xu, Lijuan Wu, Yonghong Zou, Yan Liang   +5 more
openalex   +1 more source

Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts. [PDF]

Mol Ther Methods Clin Dev
Signoria I, Zwartkruis MM, Geerlofs L, Perenthaler E, Faller KME, James R, McHale-Owen H, Green JW, Kortooms J, Snellen SH, Asselman FL, Gillingwater TH, Viero G, Wadman RI, van der Pol WL, Groen EJN.   +15 more
europepmc   +1 more source

Swallowing function in patients with spinal muscular atrophy before and after the introduction of new gene-based therapies: what has changed? [PDF]

Neurol Sci
Ruggiero M, Giannotta G, Pirani G, Saponaro F, Oliva MC, Ferrante C, Trabacca A.   +6 more
europepmc   +1 more source

The effects of disease-modifying therapies in SMA-5q type 1 and the importance of early diagnosis of the disease. [PDF]

Arq Neuropsiquiatr
Zanoteli E.
europepmc   +1 more source

The First Patient in Poland Treated for SMA with Nusinersen During Pregnancy. [PDF]

J Clin Med
Stawicka E, Skarżyńska U, Lipiec A, Mazanowska N, Stankiewicz J, Gos M, Wyszczelska-Namięta M.   +6 more
europepmc   +1 more source

Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applications. [PDF]

J Biomed Sci
Wu YF, Chen JA, Jong YJ.
europepmc   +1 more source

Switching from Nusinersen to Risdiplam: A Croatian Real-World Experience on Effectiveness and Safety [PDF]

Andrej Belančić, Tea Strbad, Marta Kučan, Dinko Vitezić   +3 more
openalex   +1 more source