Shorts and clips
These are useful discovery moments and highlights from the YouTube Shorts playlist. Full long-form conversations stay in Interviews.
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...
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.
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.
AAV vectors can work in one species and fail in another. Dr. Feng explains why the blood–brain barrier is species-specific — and what that means for brain gene therapy. Full interview on GENEration Hope.
Dr. Feng explains why SHANK3 moved into clinical trials quickly — and what it takes (money, people, focus) to translate science to patients. Full interview on GENEration Hope.
Dr. Feng on the race against time in neurodevelopmental disorders — and why earlier treatment can mean better outcomes. Full interview on GENEration Hope.
One of those moments that stops you: a student recognizes SHANK3 across the world—proof awareness is spreading.
Sue shares the quiet, hard part of stepping away—what purpose looks like after you’ve given years to a cause.
Sue explains why the pace of progress is accelerating—and why research only moves if we protect and support it.
A moment that rewired what Sue believed was possible—why hope in rare disease isn’t abstract, it’s lived. #raredisease #phelanmcdermidsyndrome #shank3 #hope #specialneedsparenting #generationhope #disability
Sue Lomas shares how rare disease organizations support each other—RSRT, Angelman, Prader-Willi and more—so no one has to build alone.
Dr. Katy Phelan on what really built the Phelan-McDermid Syndrome Foundation — families supporting each other and pushing progress forward. New interviews monthly: @GENErationHope_1 #PMSF #RareDiseaseAdvocacy #PhelanMcDermidSyndrome #GEN...
Confused by genetic testing? Dr. Katy Phelan breaks down microarray vs sequencing — and why chromosome analysis can still matter for some families. More helpful clips + monthly interviews: @GENErationHope_1 #GeneticTesting #Microarray #G...
Dr. Katy Phelan takes us back to the earliest days of identifying 22q13 deletions — and how discovery turned into collaboration, then community. Subscribe for monthly episodes. @GENErationHope_1 #22q13 #SHANK3 #Genetics #RareDisease #GEN...
Dr. Katy Phelan shares something striking: most families aren’t afraid of gene therapy — they’re worried their child won’t qualify. Follow for monthly interviews and updates. @GENErationHope_1 #GeneTherapy #ASO #RareDisease #PhelanMcDerm...
