OasisLMS

The Horizon of Prenatal Therapies for Genetic Disorders

Date of Release: February 1, 2026

Expiration Date: February 29, 2028

P.A.C.E.® credits valid through February 28, 2027

Credits offered: 1.0 AMA PRA Category 1 Credits™, P.A.C.E.®

Estimate time of completion: 1 hour

Specialized events developed by ACMG Committees.

Course Description

Learn about the evolving therapies available to treat fetuses affected by complicated genetic disorders. In this session, presenters will review the fetal presentation of lysosomal storage disorders (LSDs) and spinal muscular atrophy (SMA) and discuss novel therapeutic strategies including an ongoing phase 1 clinical trial of in utero enzyme replacement for LSDs and the prenatal use of an SMN2 splicing modifier to treat fetuses with SMA.

Course Registration Fees

Target Audience: Clinical geneticists, genetic counselors, Maternal-Fetal-Medicine physicians, researchers of all levels

Learning Objectives

At the conclusion of this session, participants should be able to:

• Recognize prenatal clinical and imaging presentations of lysosomal storage disorders (LSDs).
• Describe the scientific rationale for the ongoing Phase 1 clinical trial of in utero enzyme replacement therapy, including the potential benefits and risks.
• Describe the clinical, technical, and logistical challenges associated with delivering in utero therapy for fetuses with genetic disorders.
• Evaluate the ethical considerations, safety profile, and emerging efficacy data related to prenatal risdiplam administration for the treatment of spinal muscular atrophy in utero.

	Moderator: Roya Mostafavi, MS, CGC University of Washington School of Medicine
	Presenter: Billie Lianoglou, CGC University of California, San Francisco

Presenter: Richard Finkel, MD St. Jude Children’s Research Hospital

Planners

Henry J. Mroczkowski, MD, PhD, FACMG (Chair)

University of TN Health Science Ctr

Mari Mori, MD, MS, FACMG

The Ohio State University

Georgianne L. Arnold, MD, FACMG

University of Pittsburgh

Roya M. Mostafavi, MGC

University of Washington School of Medicine

Catherine A. Ziats, MD, FACMG

Cook Children's

Damara Ortiz, MD, FACMG

Children's Hospital of Pittsburgh of UPMC

Daniel Schecter, MD, MS

Mount Sinai School of Medicine

Staff

Jane Radford, MHA, CHCP

Director of Education - ACMG

Claudia Barnett

Senior Program Coordinator

Accredited Continuing Education Information:

AMA PRA Category 1 Credits™, P.A.C.E.®

Accreditation

The American College of Medical Genetics and Genomics is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation
The American College of Medical Genetics and Genomics designates this enduring activity for a maximum of 1 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

The American Medical Association (AMA) defines physicians as those individuals who have obtained an MD, DO, or equivalent medical degree from another country. Non-physicians may request a certificate of attendance for their participation.

ASCLS P.A.C.E.® CEU's 

ACMG is approved as a provider of continuing education programs in the clinical laboratory sciences by the American Society for Clinical Laboratory Science (ASCLS) Professional Acknowledgment for Continuing Education (P.A.C.E.®) Program.

ACMG is approved by the Florida Board of Clinical Laboratory Personnel as CE Provider (50-11878). ACMG is approved by the California Department of Health Services through the ASCLS P.A.C.E.® (275-200-26-01). This activity has been approved for 0.1 P.A.C.E.® CEU's.

Claiming your Educational Credits

This activity consists of: View content, take a post-test, the test may be taken as often as necessary to achieve a passing score of 80% or better is required to receive credit.  If you do not achieve a passing score, the program will identify which questions you answered incorrectly so that you can review the module and try again. Complete the evaluation form.

Learner Data Consent

ACMG Education reports learner data to respective agency boards and you will be asked for consent during the evaluation process. Your compliance with deadlines and completing evaluations are part of the process in meeting learner needs and ACMG’s education mission.

Technical Support:
You can reach us by email at education@acmg.net or call 301-718-9603.
Support Center Hours: Monday – Friday, 9:00 AM – 5:00 PM Eastern Time.

Accredited Continuing Education Financial Disclosure

The American College of Medical Genetics and Genomics (ACMG) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide Accredited Continuing Education (ACE) for physicians. ACMG is an organization committed to improvement of patient care and general health by the incorporation of genetics and genomics into clinical practice.

ACMG has implemented the following procedures to ensure the independence of ACE activities from commercial influence/promotional bias, the Accreditation Council for Continuing Medical Education (ACCME) requires that providers (ACMG) must be able to demonstrate that: 1) everyone in a position to control the content of an ACE activity has disclosed all financial relationships that they have had in the past 24 months with ineligible* companies; 2) ACMG has implemented a mechanism to mitigate relevant financial relationships; and 3) all relevant financial relationships with ineligible companies are disclosed to the learners before the beginning of the educational activity. The learners must also be informed if no relevant financial relationships exist.
*Ineligible companies are defined as those whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

ACMG Education Policies

Please review the policies below regarding the ACMG Education program

• Financial Disclosures and Mitigation Procedure
• ACMG Content Validation Policy
• ACMG Content Branding Policy

All of the relevant financial relationships listed for these individuals have been mitigated.

Disclaimer

ACMG educational programs are designed primarily as an educational tool for healthcare professionals who wish to increase their understanding of the application of genomic technologies to patient care. The ACMG does not endorse or recommend the use of this educational program to make patient diagnoses, particular by individuals not trained in medical genetics. Adherence to the information provided in these programs does not necessarily ensure a successful diagnostic outcome. The program should not be considered inclusive of all proper procedures and or exclusive of other procedures and that are reasonably directed at obtaining the same results. In determining the propriety of any specific procedure or, a healthcare professional should apply his or her own professional judgment to the specific clinical circumstances presented by the individual patient or specimen.

Questions regarding CE credit should be directed to education@acmg.net