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In this issue: CRISPR-Cas9 turns 10; The promise of Gene Therapy for Rare Disease and Shwachman-Diamond Syndrome (SDS) Welcome to our...

In this issue: IRDiRC Working Group Recommendations for rare disease patients; The Access to Rare Indications Act could be a game changer...

"My journey to diagnosis has been nothing short of an adventure, filled with many obstacles, hundreds of specialists, and much...