16 Jul
  • By Nic Williams
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PTC Now Enrolling for Ataluren Clinical Trial

PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren.  To ensure that participants who enroll in Study 041 get the most benefit, and to ensure their safety, there are strict criteria around who is able to enter the study. […]

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28 Jun
  • By Nic Williams
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Report from the 2018 New Direction in Biology and Disease of Skeletal Muscle Conference

The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented results of a Japanese Phase I/II dose-finding clinical study with NS-065, an exon-53 skipping drug (NS Pharma) for the treatment of Duchenne. The drug was […]

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01 Aug
  • By leilanifera
  • Cause in

Bamboo Therapeutics

Approach: Gene therapy for Duchenne
Funding Impact: Accelerated vector manufacturing capabilities
Successful Exit: Acquired by Pfizer in August 2016

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