COMPOSITIONS AND METHODS OF MODULATING SMN2 SPLICING IN SUBJECT

FIELD: medicine, pharmaceutics.SUBSTANCE: claimed group of inventions relates to field of medicine. Claimed is method of relieving at least one symptom of spinal muscular atrophy in subject, which includes introduction to subject of antisense compound, containing antisense oligonucleotide, complemen...

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Hauptverfasser: KHANG, DZHIN, SHIKHABUDDIN, LAMIJA, CHENG, SEHN, KH, RIGO, FREHNK, KREHJNER, EHDRIEHN, R, KLINGER, KEHTRIN, V, PASSINI, MARKO, A, BENNETT, S., FREHNK, KHUA, JIMIN
Format: Patent
Sprache:eng ; rus
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