South Plainfield, NJ, United States
South Plainfield, NJ, United States

PTC Therapeutics is a pharmaceutical company focused on the development of small molecule, orally administered treatments for orphan diseases. As of September 2013, PTC was headquartered at 100 Corporate Court, South Plainfield, New Jersey.They are the company responsible for the development of Ataluren.As of September 2009, PTC has entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system disease targets. Roche said use of PTC's GEMS technology for the identification of small molecules "will enable Roche to address important disease mechanisms that were intractable with conventional approaches." Wikipedia.

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Patent
Hoffmann-La Roche and PTC Therapeutics | Date: 2016-11-14

The present invention provides compounds of formula (I)


Patent
PTC Therapeutics | Date: 2017-03-31

The present invention relates to methods, compounds, and compositions for treating or preventing diseases associated with nonsense mutations in an mRNA by administering the compounds or compositions of the present invention. More particularly, the present invention relates to methods, compounds, and compositions for suppressing premature translation termination associated with a nonsense mutation in an mRNA.


Patent
PTC Therapeutics | Date: 2017-01-04

The compounds, pharmaceutical compositions, and methods of using such compounds or compositions thereof described herein are useful for treating a disease modulated by B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein expression.


Provided herein are pharmaceutical compositions, which comprise a 1,2,4-oxadiazole benzoic acid or a pharmaceutically acceptable salt thereof. Further provided herein are certain pharmaceutically acceptable salts of a 1,2,4-oxadiazole benzoic acid and methods for making the same. Further provided herein are methods of treating or preventing a disease associated with a nonsense mutation or a premature stop codon, comprising administering such pharmaceutical compositions or pharmaceutically acceptable salts to a patient having a disease associated with a nonsense mutation or a premature stop codon.


The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.


The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.


Patent
Hoffmann-La Roche and PTC Therapeutics | Date: 2017-03-22

The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.


Provided herein is a compound of formula (I):


Patent
PTC Therapeutics and Hoffmann-La Roche | Date: 2016-08-26

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.


Provided herein are pharmaceutical compositions, which comprise a 1,2,4-oxadiazole benzoic acid or a pharmaceutically acceptable salt thereof. Further provided herein are certain pharmaceutically acceptable salts of a 1,2,4-oxadiazole benzoic acid and methods for making the same. Further provided herein are methods of treating or preventing a disease associated with a nonsense mutation or a premature stop codon, comprising administering such pharmaceutical compositions or pharmaceutically acceptable salts to a patient having a disease associated with a nonsense mutation or a premature stop codon.

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