GENEration Hope Update - Sept 29, 2026

Update for the week of September 28, 2026A historic week for rare disease families — Sanfilippo syndrome just got its first-ever approved treatment, the Angelman community has a bold new gene therapy program entering the pipeline, and an ASO milestone is bringing real hope to FUS-ALS families. But approval doesn't always mean access, and we're not shying away from that conversation.\*\*In this week's update:\*\*- \[0:00] Intro & what's covered this week- \[0:45] FDA approves Fayuvi — the first-ever gene therapy for Sanfilippo Syndrome Type A- \[2:10] The $3.95M price tag: what it means for families trying to access this treatment- \[3:20] Skylark Bio enters the Angelman arena with a new UBE3A restoration program- \[4:35] Ionis reports positive Phase 3 results for ulefnersen in FUS-ALS- \[5:40] Claude AI discovers a novel enzyme system — and what that could mean for biology- \[6:30] Closing thoughts & where to find usGENEration Hope is a documentary and media platform covering rare genetic disorders, gene therapy, and the families navigating this world. Created by Ron Kleiman, a filmmaker and rare disease dad whose daughter Evie has Phelan-McDermid Syndrome. We cover what matters to families — not just investors.Subscribe and hit the bell 🔔 for weekly genetic medicine updates.\*\*Find us here:\*\*🌐 Website: https://generationhope.co📺 YouTube: @GENErationHope\_1📸 Instagram: @GenerationHopeCo\*\*Sources:\*\*🔗 FDA Approval — Fayuvi / Sanfilippo Type A: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-pediatric-patients-sanfilippo-syndrome-type🔗 Ultragenyx Price Announcement: https://www.cnbc.com/2026/09/18/ultragenyx-sets-3point95-million-price-for-rare-disease-gene-therapy.html🔗 Skylark Bio / Angelman Pipeline: \[Business Wire link]🔗 Ionis / FUS-ALS Phase 3 Results: https://ir.ionis.com/news-releases/news-release-details/ionis-announces-positive-topline-results-phase-3-fusion-study🔗 Claude AI Enzyme Discovery: https://www.anthropic.com/news/claude-discovers-novel-enzyme-system---\`\`\`0:00 Intro — A Historic Week for Rare Disease Families0:45 FDA Approves First Gene Therapy for Sanfilippo Syndrome Type A2:10 The $3.95 Million Question: Who Can Access Fayuvi?3:20 Skylark Bio Enters the Angelman Arena with New UBE3A Program4:35 Ionis Phase 3 Win: A Meaningful Milestone for FUS-ALS Families5:40 Claude AI Discovers a Novel Enzyme System6:30 Closing Thoughts — Till Next Time, Stay Hopeful\`\`\`