Online Grand Rounds and Video Library

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



Seattle Children’s is accredited by the Washington State Medical Association CME Accreditation Committee to sponsor continuing medical education for physicians.

Seattle Children’s designates this online educational activity for a maximum of 1 AMA PRA Category 1 Credit™. Physicians should only claim credit commensurate with the extent of their participation in the activity.

This activity meets the criteria for up to 1 hour of Category I CME credit to satisfy the relicensure requirements of the Washington State Medical Quality Assurance Commission.

Videos are available for only one year after posting.

If you wish to receive Category I CME credit for viewing this Provider Grand Rounds session, please complete the evaluation form. Upon completion of the form you will receive an auto-reply email that will serve as the only confirmation of your CME credit. Please keep the email for your records.