Exonics to develop a therapeutic approach to correct underlying genetic mutations in Duchenne, a degenerative genetic disorder without effective treatment
options to halt progression of disease.
NEWPORT BEACH, Calif., February 27, 2017 – CureDuchenne Ventures, a subsidiary of the nonprofit CureDuchenne that funds research to find a cure for Duchenne muscular dystrophy, announced today that they […]
Haydee Bustos has a passion for helping kids, and through her experience as a physical therapist, she discovered an even deeper desire to help kids with Duchenne muscular dystrophy. […]
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
