METHODS FOR TREATING SPINAL MUSCULAR ATROPHY

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United States of America Patent

APP PUB NO 20150283115A1
SERIAL NO

14621051

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Abstract

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Described herein are methods for the identification or validation of compounds capable of causing ribosomal frameshifting and the use of said compounds to produce a stabilized SMNΔEx7 protein and treat Spinal Muscular Dystrophy.

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Patent Owner(s)

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PTC THERAPEUTICS INC100 CORPORATE COURT SOUTH PLAINFIELD NJ 07080-2449

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Naryshkin, Nikolai A East Brunswick, US 14 108
Paushkin, Sergey V Belle Mead, US 8 117
Welch, Ellen Califon, US 37 313

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