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Clinical Implementation of Gene-Targeted Therapy Including Gene Replacement Therapy for Neuromuscular Diseases

Activity Snapshot

Activity Type: 
On-Demand Webinar
Release Date: 
Friday, October 25, 2019
Expiration Date: 
Monday, October 24, 2022
Sponsor Information: 

This program is supported in part by an independent medical education grant from AveXis Inc.

Faculty

Diana Bharucha-Goebel, MD
Diana Bharucha-Goebel, MD
Pediatric Neuromuscular Neurologist, Assistant Professor
Children's National Hospital, National Institute of Health/ NINDS
Clinical Implementation of Gene-Targeted Therapy Including Gene Replacement Therapy for Neuromuscular Diseases
Activity Overview: 

There are a growing number of treatments that target the underlying genetic cause of neuromuscular diseases. This webinar will examine two approaches, exon skipping and gene replacement using viral vectors, being used to treat spinal muscular atrophy and Duchenne muscular dystrophy. Topics addressed include mechanism of action, technical issues, practical considerations and future directions.

Learning Objectives: 
  • Discuss the biological rationale for using gene-targeted therapies to treat certain neuromuscular diseases and review the importance of early diagnosis and intervention in the context of the pathophysiology of these diseases
  • Provide education about the development of gene-targeted therapies, including gene-replacement therapy, as treatments for neuromuscular diseases
  • Discuss practical considerations when using these therapies in the clinic, including patient selection and eligibility, handling of the therapy, administration, and safety and monitoring
  • Discuss ways to address questions from patients and caregivers about gene-targeted therapies and setting expectations for treatment
Accreditation and Designation Statement: 

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of PeerPoint Medical Education Institute, LLC and Muscular Dystrophy Association. The PeerPoint Medical Education Institute, LLC is accredited by the ACCME to provide continuing medical education for physicians.

The PeerPoint Medical Education Institute, LLC designates this Enduring Material activity for a maximum of .75 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Release Date: October 25, 2019, Termination Date: October 25, 2022

How to Obtain CME credits : 

Once you have viewed the program, MDA will share back with you instructions for how to obtain your CME credits. If you have questions on this process, please reach out to cmemanagement@mdausa.org.

Faculty Disclosures: 

The following presenters, planners, editors, or staff have relevant financial relationships to disclose:

“I, or an immediate family member, have at present or have had within the last 12 months, the following affiliation with one or more organizations that could be perceived as a real or apparent conflict of interest in context to the design, implementation, presentation, evaluation, etc. of CME Activities."

Diana Bharucha-Goebel:

Gene Therapy Network (Avexis): Educational Content Consultant; Consulting Fees

All other presenters, planners, editors, or staff reported no relevant relationships with the following statement:

“I, or an immediate family member, do not have at present, or within the last 12 months, nor anticipate having financial interest, arrangement or affiliation with any organizations that could be perceived as a real or apparent conflict of interest in context to the design, implementation, presentation, evaluation, etc. of CME Activities.”

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