Advancements in Understanding Facioscapulohumeral Muscular Dystrophy (FSHD) and What Clinically Meaningful Outcomes Look Like

Program Introduction

Delve into the cutting-edge advancements in Facioscapulohumeral Muscular Dystrophy (FSHD) alongside the dynamic duo of Dr. Leo H. Wang and Dr. Maya N. Hatch at the 2024 MDA Clinical & Scientific Conference Summit. Positioned as the third most prevalent muscular dystrophy, FSHD stands as a genetic enigma with intricate roots, showcasing a progressive saga of asymmetrical muscle weakness. Initially targeting the facial and shoulder muscles before spreading its grip onto the extremities, this disorder's clinical narrative presents a formidable challenge in quantifying outcomes. It's an imperative moment to unearth dependable and sensitive metrics for clinical trials, navigating the labyrinthine landscape of FSHD. Acquire invaluable insights into mastering daily functionalities, tackling complications head-on, and remain abreast of the latest breakthroughs in establishing rock-solid measures for gauging progress.

Learning Objectives

  • Evaluate the use of RWS as an outcome measure for FSHD clinical trials and translate changes in RWS to clinically meaningful outcomes
  • Discuss the mechanism of FSHD disease that leads to challenges in DUX4 biomarker detection

Available Credit

  • 1.25 AMA PRA Category 1 CreditTM
  • 1.25 Participation

Faculty Affiliation

Leo H. Wang, MD, PhD
Professor of Neurology
Seattle Wellstone Center
Seattle, WA

Maya N. Hatch, PhD
Clinical Researcher
Department of Physical Medicine & Rehabilitation
UC Irvine Health, Irvine, CA USA

Financial Disclosure

The CME Institute adheres to the Standards for Integrity and Independence in Accredited Continuing Education of the Accreditation Council for Continuing Medical Education (ACCME). Any individuals in a position to control the content of a continuing education activity, including faculty, content developers, reviewers, staff, and others, are required to disclose to learners the presence or absence of any relevant financial relationships with an ACCME-defined ineligible company within the preceding 24 months of the activity. The ACCME defines an “ineligible company” as one whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.

The CME Institute has mitigated all relevant conflicts of interest prior to the commencement of the activity. None of the individuals involved in the content have relevant financial relationships with ineligible companies except the following:

Dr M. N. Hatch has received consulting fees from Bioniks and Fulcrum Therapeutics; and has received advisory board fees from Fulcrum Therapeutics.

Dr L. H. Wang has received consulting fees from Fulcrum Therapeutics; and has received grant/research support from Avidity Bioscience and Fulcrum Therapeutics. 

Dr. E. Ciafaloni, peer reviewer, has received grant/research support from Sarepta; and has received advisory board fees from Alexion, Pfizer, Sarepta, Pfizer, and UCB.

None of the other faculty, planners, reviewers, and CME Institute staff for this educational activity have relevant financial relationships with ineligible companies to disclose. All relevant financial relationships have been mitigated.

Support Statement

Supported by an educational grant from Fulcrum Therapeutics

Release and Expiration Dates

This CME activity took place on Tuesday, March 5, 2024 and is eligible for AMA PRA Category 1 Credit™ only to those who attended the activity in person or virtually during the 2024 MDA Clinical and Scientific Conference.

Accreditation Statement

The CME Institute of Physicians Postgraduate Press, Inc., is accredited by the Accreditation Council for Continuing Medical Education to provide continuing medical education for physicians.

Credit Designation

The CME Institute of Physicians Postgraduate Press, Inc., designates this enduring material for a maximum of 1.25 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Note: The American Nurses Credentialing Center (ANCC) and the American Academy of Physician Assistants (AAPA) accept certificates of participation for educational activities certified for AMA PRA Category 1 Credit™ from organizations accredited by the ACCME.

To obtain credit for this activity, study the material and complete the Evaluation.