Eszter Hars, Ph.D., President and CEO, SDS AllianceJul 10, 2022SDS Science SnapshotsSDS & Science Snapshots (2022-07-10)In this issue: CRISPR-Cas9 turns 10; The promise of Gene Therapy for Rare Disease and Shwachman-Diamond Syndrome (SDS) Welcome to our...
Eszter Hars, Ph.D., President and CEO, SDS AllianceJul 3, 2022SDS Science SnapshotsSDS & Science Snapshots (2022-07-03)In this issue: IRDiRC Working Group Recommendations for rare disease patients; The Access to Rare Indications Act could be a game changer...
Eszter Hars, Ph.D., President and CEO, SDS AllianceJun 25, 2022SDS Science SnapshotsSDS & Science Snapshots (2022-06-25)In this issue: Rare-X report provides update on the true number of rare diseases; new review article on bone marrow failure disorders by...