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It summarizes FSHD as a progressive, disabling muscular dystrophy, detailing key symptom impacts on mobility, pain, function, independence, and psychological wellbeing, and explains how survey results informed the agenda. The report also outlines treatment limitations and unmet needs for future therapy development.",{"@graph":69,"@context":123},[70,84,106],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/voice-of-the-patient-report-externally-led-patient-focused-drug-development-meeting/296547/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/voice-of-the-patient-report-externally-led-patient-focused-drug-development-meeting/296547.png","ImageObject",300,407,{"name":92,"@type":93},"Jacob","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-23","2026-09-18",true,{"@type":102,"interactionType":103,"userInteractionCount":105},"InteractionCounter",{"@type":104},"ViewAction",12,{"@type":107,"mainEntity":108},"FAQPage",[109,115,119],{"name":110,"@type":111,"acceptedAnswer":112},"What is the purpose of the Voice of the Patient meeting for FSHD?","Question",{"text":113,"@type":114},"The meeting enables the FDA and other drug development stakeholders to hear directly from patients and caregivers about their journey with FSHD, including symptom impact, current treatments, and unmet needs.","Answer",{"name":116,"@type":111,"acceptedAnswer":117},"How does the report describe facioscapulohumeral muscular dystrophy (FSHD)?",{"text":118,"@type":114},"FSHD is presented as a serious, rare, heterogeneous, progressive, and disabling muscular dystrophy affecting stereotypical muscle groups such as the face, shoulder blades, and upper arm, with possible involvement of lower limb and abdominal muscles.",{"name":120,"@type":111,"acceptedAnswer":121},"What evidence was used to shape the meeting?",{"text":122,"@type":114},"Prior to the meeting, nearly 600 people worldwide completed a survey on how FSHD affects their lives, including inputs from individuals living with FSHD and caregivers, and those data were used to shape the agenda and discussions.","https://schema.org",{"og:url":83,"og:type":125,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":127,"canonical":83},"index,follow",{"doc_id":129,"site_id":62},296547,1790188828,{"code":4,"msg":5,"data":132},{"doc_id":129,"user_id":133,"nickname":92,"user_avatar":134,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":135,"file_id":136,"file_url":137,"file_type":138,"file_size":139,"view_count":105,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":140,"language":141,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":142,"faqs":143,"seo_title":144,"seo_description":67,"update_tm":145,"read_time":146},962084931830,"https://ap-avatar.wpscdn.com/davatar_a8503ba1806abce46bf441b54a3ca4cd","Facioscapulohumeral  \nMuscular Dystrophy (FSHD)  \nVOICE OF THE PATIENT REPORT  \nExternally Led Patient-Focused Drug Development Meeting  \nThis report is dedicated to the individuals who courageously shared their stories.  \nAUTHORS: Jamshid Arjomand, PhD, Chief Science Officer, FSHD Society; Lisa Bain, Medical Writer; June Kinoshita, Director of Research and Patient Engagement, FSHD Society; Mark Stone, President and Chief Executive Officer, FSHD Society  \nFUNDING AND ADVOCACY PARTNERS: Friends of FSH Research, Fulcrum Therapeutics, Muscular Dystrophy Association, Optum  \nCONSULTING PARTNERS: James E. Valentine, JD MHS, and Larry Bauer, RN MA, Hyman, Phelps & McNamara, P. C.  \nCLINICAL ADVISOR: Kathryn Wagner, MD PhD, Kennedy Krieger Institute and Johns Hopkins School of Medicine  \nDISCLOSURES: Dr. Wagner is a consultant for Dynacure, PTC Therapeutics, Casma Therapeutics, Asklepios BioPharmaceutical, Inc., Santhera, Regenxbio, and Sarepta Therapeutics, and principal investigator for clinical trials sponsored by Pfizer, Sarepta Therapeutics, Catabasis, Fulcrum, and  \nPTC Therapeutics. Hyman, Phelps & McNamara, P. C., is a law firm that represents sponsors who are developing drugs for rare diseases as well as rare disease patient advocacy organizations, including the FSHD Society. No company was involved in the planning and coordination of the meeting.  \nACKNOWLEDGMENT: We thank William Lewallen, Meghana Chalasani, Michelle Campbell, PhD, and their colleagues at the FDA’s Center for Drug Evaluation and Research for their guidance in planning the EL-PFDD.  \nPUBLICATION DATE: November 5, 2020  \nREVISIONS AND MODIFICATIONS: This document has not been revised and/or modified in anyway after the version date listed above and on the cover page. The submitters have all necessary permissions to submit this external resource, and linking from the FDA website will not violate the proprietary rights of others.  \nPOINT OF CONTACT: June Kinoshita,[june.kinoshita@fshdsociety.org](june.kinoshita@fshdsociety.org)  \nTABLE OF CONTENTS   \n1. Report summary  ................................................................ 1  \n2. Introduction  .................................................................... 3  \n3. Overview of FSHD and its management  ............................................. 4  \n4. Meeting overview  ................................................................ 6  \n5. Topic 1: Symptoms of FSHD and daily impacts that matter most to patients  ............. 10  \nMuscle weakness and other symptoms  ............................................ 10  \nConsequences of FS HD symptoms  ................................................ 14  \nImpact on daily life  .............................................................. 19  \nConcerns about the future  ........................................................ 24  \n6. Topic 2: Managing FS HD  ......................................................... 27  \nCurrently available treatments and their effectiveness  ................................ 27  \nHopes and expectations for future treatment  ........................................ 37  \n7. Incorporating patient input into a benefit-risk framework for FSHD  ..................... 40  \n8. Conclusions  ................................................................... 43  \n9. Appendix  .................................................................. 44–138  \nI  . Testimony from live webcast on June 29  ........................................ 44  \nII  . Panelists’ written testimony  ................................................... 67  \nIII  . Testimony submitted by email  ................................................. 87  \nIV . Live-polling questions  ....................................................... 129  \nV . Pre-meeting survey questions  ................................................ 134  \nF S H D S O C I E T Y | V O I C E O F T H E P A T I E N T R E P O R T  \n1  \n1. Report summary  \nFacioscapulohumeral muscular dystrophy","cbCaiopo5eVzr6bl","https://ap.wps.com/l/cbCaiopo5eVzr6bl","pdf",4243968,140,"English","# Report summary\n# Introduction\n# Overview of FSHD and its management\n# Meeting overview\n# Topic 1: Symptoms of FSHD and daily impacts that matter most to patients\n## Muscle weakness and other symptoms\n## Consequences of FSHD symptoms\n## Impact on daily life\n## Concerns about the future\n# Topic 2: Managing FSHD\n## Currently available treatments and their effectiveness\n## Hopes and expectations for future treatment\n# Incorporating patient input into a benefit-risk framework for FSHD\n# Conclusions\n# Appendix","[{\"question\":\"What is the purpose of the Voice of the Patient meeting for FSHD?\",\"answer\":\"The meeting enables the FDA and other drug development stakeholders to hear directly from patients and caregivers about their journey with FSHD, including symptom impact, current treatments, and unmet needs.\"},{\"question\":\"How does the report describe facioscapulohumeral muscular dystrophy (FSHD)?\",\"answer\":\"FSHD is presented as a serious, rare, heterogeneous, progressive, and disabling muscular dystrophy affecting stereotypical muscle groups such as the face, shoulder blades, and upper arm, with possible involvement of lower limb and abdominal muscles.\"},{\"question\":\"What evidence was used to shape the meeting?\",\"answer\":\"Prior to the meeting, nearly 600 people worldwide completed a survey on how FSHD affects their lives, including inputs from individuals living with FSHD and caregivers, and those data were used to shape the agenda and discussions.\"}]","Voice of the Patient Report - Externally Led Patient-Focused Drug Development Meeting | PDF",1789733715,353]