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Dr. Eszter Hars (SDS Alliance president and CEO) joined the FDA CBER OTAT Patient-Focused Drug Development Listening Meeting.

In this issue: What is neutropenia and how does it happen in SDS; Study highlights the multibillion dollar burden of rare disease Welcome...

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