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Date: August 21, 2025
Time: 11-12pm EDT / 5-6pm CEST
Title: The importance of involving patients in therapy development; Lessons learned from Duchenne exon skipping
Description:
Duchenne muscular dystrophy is a progressive muscle wasting disease caused by lack of dystrophin protein. The exon skipping approach uses antisense oligonucleotides to manipulate splicing, such that the transcript open reading frame is restored and Duchenne patients can make partially functional dystrophins such as found in less severely progressive Becker muscular dystrophy patients.
In this interactive lecture I will take the audience through several decision making steps of preclinical and clinical development of the exon skipping approach for Duchenne muscular dystrophy I was involved in.
Disclaimer and expectation management: There will be many similarities between this lecture and my keynote lecture at the OTS meeting in Montreal.
