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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...

In this issue: What is Ataluren and what does recent research show about its potential use in treating individuals with SDS in the...

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