16 Jul
  • By CureDuchenne User
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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
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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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15 Feb
  • By leilanifera
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Sarepta Therapeutics

Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne

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15 Feb
  • By leilanifera
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Prosensa/BioMarin

Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015

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15 Feb
  • By leilanifera
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Nationwide Children’s Hospital

Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys

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