Online Grand Rounds and Video Library

Gene-Targeted Therapy for Neurodevelopmental and Neurodegenerative Disorders: The Example of Spinal Muscular Atrophy

Objectives and Disclosures

Participants will be able to:

  1. analyze rare neurogenetic diseases and spinal muscular atrophy as an example
  2. assess advances in the treatment of patients with SMA: Discovery, Innovation, Disruption and Transformation
  3. use the example of a case with SMA to learn about prenatal therapy for genetic disorders

Planners disclose they have no relevant financial relationships. Speaker discloses: Intends to reference off-label or investigational use of Nusinersen, onasemnogene, abeparvovec, and risdiplam; Research support from AveXis, Biogen, Capricor, Catabasis, Ionis, Italfarmaco, PTC Therapeutics, ReveeraGen, Roche, Sarepta, Santhera, Scholar Rock, Summit; On advisory boards of AveXis/Novartis, Biogen, Genentech/Roche, Ionis, Sarepta; DSMB Member. All relevant financial relationships have been mitigated.


Richard S. Finkel, MD
Director, Center for Experimental Neurotherapeutics, St. Jude Children’s Research Hospital, Memphis, TN



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.