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In this issue: Breaking news! First CRISPR-based therapy approved by the FDA for treatment of a genetic blood disorder - sickle cell...

Shwachman-Diamond Syndrome receives new ICD-10 Diagnostic Code, Paving the Way For Improved Patient Care and New Treatment Options. Code...

In this issue: New proof-of-concept article on RNA Therapeutics and Base/Prime Editing for SDS; And, a review of diagnostic testing for...