Gene Therapy: Application to Clinical Practice

Published by Pediatric Pharmacy Association


This presentation will review the basics of gene therapy including important considerations for hospitals that will be handling these medications. The application to clinical practice will focus on spinal muscular atrophy and the gene therapy for it as well as our institution's specific experience with these patients.

Learning Objectives for Pharmacists
At the end of this session, participants will be able to:
1. Identify the general principles of gene modifying/replacement therapies and application to the treatment of rare disorders
2. Describe the safe handling & dispensing implications of gene therapy agents
3. Review the current marketed agents nusinersen and omnasemnogene abeparvovex-xioi and their application in spinal muscular atrophy
4. Discuss other gene therapy agents in the pipeline

ACPE logo PPAG has accredited this knowledge-based webinar for 1.0 contact hours (0.10 CEUs) of continuing education credit for pharmacists. UAN #:0180-0000-20-007-H01-P.

The Pediatric Pharmacy Association is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. In order to receive credit, participants must register for the program, attend the program, and fill out a post-program survey. Webinar programs are 100% supported through program registration fees and PPA membership dues, and is not supported by Industry.

Group Pricing for CE only:
Groups will need to email Lauren Henretty at their attendance list with each participants name, email, NABP# and DOB in order to receive credit.

James Dice, PharmD, BCPPS
Clinical Specialist - P/T, Former Pharmacy Director - Children's Hospital of The King's Daughters
Dr. Dice has been an established neonatal, pediatric clinical practitioner and pediatric pharmacy practice manager & leader. Dr. Dice recently retired from the Director of Pharmacy position at the Children’s Hospital of The King’s Daughters after 16...More
Christina Hellauer, PharmD
Outpatient Clinical Pharmacy Specialist - Children's Hospital of The King's Daughters
Dr. Hellauer is currently the Outpatient Clinical Pharmacy Specialist at Children’s Hospital of The King’s Daughters, where she is primarily responsible for the outpatient infusion center, outpatient renal services including Dialysis and the Renal...More

Originally Published

July 22, 2020

Program Titles and Supporting Materials

This program contains the following components:

  • Gene Therapy: Application to Clinical Practice - Video
  • Gene Therapy: Application to Clinical Practice - Paper

How To Attend

Join the self-paced program from your office, home, or hotel room using a computer and high speed internet connection. You may start and stop the program at your convenience, continue where you left off, and review supporting materials as often as you like. Please note: Internet Explorer is no longer a supported browser. We recommend using Google Chrome, Mozilla Firefox or Safari for best results.

Technical Requirements

You may access this course on a computer or mobile device with high speed internet (iPhones require iOS 10 or higher). Recommended browsers are Google Chrome or Mozilla Firefox.


If applicable, you may obtain credit in multiple jurisdictions simultaneously for this program (see pending/approved list below). If electing credit for this program, registrants in jurisdictions not listed below will receive a Certificate of Completion that may or may not meet credit requirements in other jurisdictions. Where applicable, credit will be only awarded to a paid registrant completing all the requirements of the program as determined by the selected accreditation authority.

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