Results 221 to 230 of about 7,776 (285)

MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target. [PDF]

Mol Ther Nucleic Acids
Chen JA.
europepmc   +1 more source

Spinal presentations in children with type 1 spinal muscular atrophy on nusinersen treatment across the SMA-REACH UK network: a retrospective national observational study. [PDF]

BMJ Open
Abbott L, Main M, Wolfe A, Rohwer A, Baranello G, Munot P, Manzur A, Muntoni F, Scoto M, SMA-REACH UK Network Physiotherapists.   +9 more
europepmc   +1 more source

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series. [PDF]

BMC Neurol
Svoboda MD, Kuntz N, Leon-Astudillo C, Byrne BJ, Krueger J, Kwon JM, Sieburg C, Castro D.   +7 more
europepmc   +1 more source

Effect of Nusinersen on Respiratory Muscle Function in Different Subtypes of Type 1 Spinal Muscular Atrophy [PDF]

, 2019
Alberti, K, Aliverti, A, Baranello, G, LoMauro, A, Masson, R, Mastella, C   +5 more
core  

RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System. [PDF]

Biomol Ther (Seoul)
Ertural B, Çiçek BN, Kurnaz IA.
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

Patient-specific responses to <i>SMN2</i> 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

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

New England Journal of Medicine, 2017
Richard S Finkel, Eugenio Mercuri, Basil T Darras   +2 more
exaly   +2 more sources