Comment from CureDuchenne - Newport Beach, CA
AnonymousSupportAdvocacy
Summary: CureDuchenne, a patient advocacy organization for Duchenne muscular dystrophy, strongly supports the FDA's draft guidance on leveraging prior knowledge for genome editing therapies. They argue that streamlining development through shared data and platform experience is critical for addressing urgent unmet needs in rapidly progressive pediatric diseases.
CureDuchenne Comments_See attached file(s)