15 Feb
  • By leilanifera
  • Cause in

Sarepta Therapeutics

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

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15 Feb
  • By leilanifera
  • Cause in

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
  • Cause in

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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