The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 04, 2019
Filed:
Sep. 04, 2017
Ptc Therapeutics, Inc., South Plainfield, NJ (US);
F. Hoffmann-la Roche Ag, Basel, CH;
Matthew G. Woll, Dunellen, NJ (US);
Guangming Chen, Bridgewater, NJ (US);
Soongyu Choi, New York, NY (US);
Amal Dakka, Whitehouse Station, NJ (US);
Song Huang, San Leandro, CA (US);
Gary Mitchell Karp, Princeton Junction, NJ (US);
Chang-Sun Lee, Belle Mead, NJ (US);
Chunshi Li, East Brunswick, NJ (US);
Jana Narasimhan, Scotch Plains, NJ (US);
Nikolai Naryshkin, East Brunswick, NJ (US);
Sergey Paushkin, Belle Mead, NJ (US);
Hongyan Qi, Plainsboro, NJ (US);
Anthony A. Turpoff, Hillsborough, NJ (US);
Marla L. Weetall, Morristown, NJ (US);
Ellen Welch, Califon, NJ (US);
Tianle Yang, Mountainside, NJ (US);
Nanjing Zhang, Princeton, NJ (US);
Xiaoyan Zhang, Belle Mead, NJ (US);
Xin Zhao, Belle Mead, NJ (US);
Emmanuel Pinard, Linsdort, FR;
Hasane Ratni, Habsheim, FR;
PTC Therapeutics, Inc., South Plainfield, NJ (US);
F. Hoffmann-La Roche, Basel, CH;
Abstract
Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.