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List of works by Kenneth H. Fischbeck

Combinatorial treatment for spinal muscular atrophy: An Editorial for 'Combined treatment with the histone deacetylase inhibitor LBH589 and a splice-switch antisense oligonucleotide enhances SMN2 splicing and SMN expression in Spinal Muscular Atroph

scientific article published on 14 February 2020

Hereditary spastic paraplegia type 43 (SPG43) is caused by mutation in C19orf12

scientific article

ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice

scientific article

Mutant dynactin in motor neuron disease

scientific article published on 10 March 2003

Mutation in CPT1C Associated With Pure Autosomal Dominant Spastic Paraplegia

scientific article

SMN Modulator ML372 Increases SMN Protein Abundance, Body Weight, Lifespan, and Rescues Motor Function in SMNΔ7 SMA Mice

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