Rare Disease
Science Needs Defenders
Sue explains why the pace of progress is accelerating—and why research only moves if we protect and support it.
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Gene TherapyClinical TrialsRare Disease
Gene Therapy Weekly: CRISPR's Biggest Trial Result Yet | GENEration Hope
Update for the week of April 27, 2026 A CRISPR-based treatment just delivered pivotal Phase 3 results for a rare and debilitating disease — and for rare disease families, this is a milestone worth understanding. This week, Ron breaks dow...
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Rare Disease
“If We Solve This… I’ll Be the Happiest Person” | Guoping Feng
Dr. Feng talks about the reality families live with — and what it would mean if we could truly treat severe neurodevelopmental disorders. Full interview on GENEration Hope.
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Rare Disease
“It’s Technical — We Can Solve It” | Why He’s Optimistic
Dr. Feng explains why many monogenic neurodevelopmental disorders feel solvable now — if we put enough people and resources behind them. Full interview on GENEration Hope.