Blog Archives

Catching Up With 3 Rare Disease Families

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Eliza’s Journey: Part 2

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Eliza’s Journey: Part 1

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A Checklist for Gene Therapy From the UK Cystic Fibrosis Trial

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Rare Disease Day 2014: A Parent Fights to Cure Blindness

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Gene Therapy News: Brain, Skin, Eye

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A Little Girl With Giant Axons, a Deranged Cytoskeleton, and Someday Gene Therapy

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Rare Disease Day: What 5 Kids with Low Vision CAN Do

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Another Bump in the Road to Gene Therapy?

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Gene Therapy for Canavan Disease: Max’s Story

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