Cart
×
Summit
Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial
Sarepta Therapeutics
Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne
PTC Therapeutics
Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU
Prosensa/BioMarin
Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015
cTAP
Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design
Nationwide Children’s Hospital
Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys
An Open Letter to the Duchenne Community
CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: […]
An Open Letter to the Duchenne Community
CureDuchenne is sharing an expanded and updated open letter to the Duchenne community from Marathon Pharmaceuticals. An Open Letter to the Duchenne Community Dear Members of the Duchenne Community: […]
FDA Approves EMFLAZA (deflazacort) … Our Experience With Deflazacort
We are delighted that the U.S. Food and Drug Administration (FDA) has approved EMFLAZA (deflazacort) for the treatment of Duchenne. This is wonderful news for the Duchenne community to […]