28 Jun
  • By Nic Williams
  • Cause in

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
  • Cause in

cTAP

Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design

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