TRiNDS
Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective
Approach: Neuromuscular focused Contract Research Organization
Funding Impact: Clinical trial support for Biotech/Pharma from a patient-centric perspective
Approach: Utrophin upregulation
Funding Impact: Enabled initiation of Phase 2 clinical trial
Approach: Exon skipping
Funding Impact: First drug approved by FDA to treat Duchenne
Approach: Stop codon read-through
Funding Impact: Conditional approval of Translanrna in EU
Approach: Exon skipping
Funding Impact: Helped support clinical development of exon skipping drugs
Successful Exit: Prosensa was acquired by BioMarin in 2015
Approach: Statistical analysis of natural history studies
Funding Impact: Collaboration amongst biotech/pharma to share data and enrich clinical trial design
Approach: Exon skipping (Duplication exon 2 mutations)
Funding Impact: Advanced pre-clinical IND package enabling clinical trial for Duchenne boys
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: […]
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: […]