CRISPR Genome Editing for Correction of Rare Disease Pathogenic Variants
Objectives and Disclosures
- Participants will be able to:
- Develop a plan for diagnostic confirmation once notified that a neonate has an abnormal newborn screening result for Pompe disease.
- Implement a multidisciplinary treatment plan for a neonate who has been confirmed to have a diagnosis of Pompe disease.
- Contrast the strengths and limitations of current standards-of-care therapies for Pompe disease with future therapies in development, such as CRISPR genome editing.
Planners disclose they have no relevant financial relationships. Speaker discloses the following relationships: Biomarin Pharmaceuticals: Grant/Research Support, Consultant, Stocks/Stock Options, and Honoraria. Takeda: Consultant and Honoraria.
All relevant financial relationships have been mitigated.
Raymond Wang, MD
Director of the Multidisciplinary Lysosomal Storage Disorder Program, Children’s Hospital of Orange, CA
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