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JCI Insight|June 10, 2020
Minor snRNA gene delivery improves the loss of proprioceptive synapses on SMA motor neuronsErkan Y Osman, Meaghan Van Alstyne, Pei-Fen Yen, et al.
Biorxiv : the Preprint Server for Biology|April 16, 2025
A chemical screen identifies p38 MAPK inhibition as a candidate neuroprotective strategy for combinatorial SMA therapyMaria J Carlini, Jorge Espinoza-Derout, Meaghan Van Alstyne, et al.
Experimental Neurology|February 20, 2016
Protective effects of butyrate-based compounds on a mouse model for spinal muscular atrophyMatthew E R Butchbach, Casey J Lumpkin, Ashlee W Harris, et al.
Communications Medicine|February 16, 2026
Nusinersen rescues taurine deficiency in patients with type 1 Spinal Muscular AtrophyRaffaella di Vito, Amber Hassan, Tommaso Nuzzo, et al.
Neurobiology of Disease|August 2, 2025
Nusinersen corrects L-arginine deficiency in the cerebrospinal fluid of patients with severe spinal muscular atrophyAmber Hassan, Raffaella di Vito, Anna Caretto, et al.
Scientific Reports|June 26, 2023
Evaluation of the orally bioavailable 4-phenylbutyrate-tethered trichostatin A analogue AR42 in models of spinal muscular atrophyCasey J Lumpkin, Ashlee W Harris, Andrew J Connell, et al.
Nature Chemical Biology|February 20, 2019
FTO controls reversible m<sup>6</sup>Am RNA methylation during snRNA biogenesisJan Mauer, Miriam Sindelar, Vladimir Despic, et al.
Biorxiv : the Preprint Server for Biology|September 11, 2024
Synaptic imbalance and increased inhibition impair motor function in SMAEmily V Fletcher, Joshua I Chalif, Travis M Rotterman, et al.
EMBO Molecular Medicine|September 9, 2025
Identification of p38 MAPK inhibition as a neuroprotective strategy for combinatorial SMA therapyMaria J Carlini, Jorge Espinoza-Derout, Meaghan Van Alstyne, et al.
Cell Reports|December 28, 2017
Converging Mechanisms of p53 Activation Drive Motor Neuron Degeneration in Spinal Muscular AtrophyChristian M Simon, Ya Dai, Meaghan Van Alstyne, et al.
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