I say «curious» because Marathon came under intense scrutiny for its initial plans to hike the drug's price to $ 89,000 per year even though it's
available for a mere pittance in other countries (and doesn't actually address the root causes of
Duchenne, but rather just some of its muscle - wasting symptoms).
The CRISPR / Cas9 platform for
Duchenne developed by the UCLA scientists is not yet
available in clinical trials and has not been approved by the FDA for use in humans.