Results 111 to 120 of about 2,169 (197)

Synergistic Effect of an Antisense Oligonucleotide and Small Molecule on Splicing Correction of the Spinal Muscular Atrophy Gene

Neuroscience Insights
Spinal muscular atrophy (SMA) is treated by increasing the level of Survival Motor Neuron (SMN) protein through correction of SMN2 exon 7 skipping or exogenous expression of SMN through gene therapy.
Eric W Ottesen, Ravindra N Singh
doaj   +1 more source

Evaluation of disease progression and response to therapy in a cohort of late childhood/adulthood SMA patients: Is there room for new markers? [PDF]

, 2023
LUPICA, Antonino
core  

Spinal muscular atrophy:From approved therapies to future therapeutic targets for personalized medicine [PDF]

, 2021
Chaytow, Helena, Faller, Kiterie, Gillingwater, Thomas H, Huang, Yu-ting   +3 more
core   +1 more source

Risdiplam therapy in adult patients with Spinal muscular Atrophy (SMA). A 24-month-real-world experience at a single muscular centre. [PDF]

Acta Myol
Cesarone E, Picillo E, Scutifero M, Passamano L, Nigro V.   +4 more
europepmc   +1 more source

Risdiplam for spinal muscular atrophy

Australian Prescriber, 2022
openaire   +2 more sources

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series. [PDF]

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

Evaluating effects of risdiplam in adults with spinal muscular atrophy: a monocentric study. [PDF]

ERJ Open Res
Crescimanno G, Alonge P, Lupica A, Tomasello V, Di Stefano V, Brighina F, Marrone O.   +6 more
europepmc   +1 more source

Neurodevelopmental Comorbidities in Early-Onset 5q-SMA Treated with Disease-Modifying Therapies: A Scoping Review [PDF]

Recent studies have reported possible neurodevelopmental comorbidities in some early onset 5q-spinal muscular atrophy (5q-SMA) patients treated with disease-modifying therapies (DMT).
Konop, Carson
core   +1 more source

Matched-pair analysis of motor outcomes in adults with spinal muscular atrophy on nusinersen vs. risdiplam. [PDF]

J Neurol
Neuhoff S, Stolte B, Lipka J, Schlag M, Pul R, Schmitt LI, Leo M, Skuljec J, Deuschl C, Forsting M, Kleinschnitz C, Hagenacker T.   +11 more
europepmc   +1 more source

Treatment evolution in spinal muscular atrophy: insights from the SMArtCARE registry. [PDF]

Brain
Voigt-Müller C, Pfaffenlehner M, Bernert G, Cetin H, Hagenacker T, Kölbel H, Lochmüller H, Pfeuffer C, Vill K, Walter MC, Kirschner J, Pechmann A, SMArtCARE study group .   +12 more
europepmc   +1 more source