METHODS OF TREATING OR PREVENTING SPINAL MUSCULAR ATROPHY

Abstract: featured are methods for the treatment or prevention of spinal muscular atrophy. effective dosage regimens are specified. biomarkers and kits are also provided.

Inventor(s): Kenneth Swope Loveday, Fengju Judy Bai, Lilly East, Wildon R. Farwell

CPC Classification: C12N15/1136 (Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants )})

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