Facioscapulohumeral Muscular Dystrophy
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy [Phase 1, Phase 2]
A first-in-human Phase 1/2 study of EPI-321 for facioscapulohumeral muscular dystrophy is currently recruiting, sponsored by Epicrispr Biote…
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: Amino Acids and Exercise in FSHD [NA]
A clinical trial is evaluating the effects of amino acids and exercise in facioscapulohumeral muscular dystrophy (FSHD); recruitment is ongo…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
A clinical trial update describes a project to identify and address barriers to drug development for Facioscapulohumeral Muscular Dystrophy.…
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy [Phase 2]
This document provides an update on Hoffmann-La Roche's Phase 2 study of RO7204239 for facioscapulohumeral muscular dystrophy, currently act…
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: Walking ANalysis Interest in Persons wiTh facioscapulohumEral Muscular Dystrophies [NA]
The document reports that a clinical trial studying walking analysis in adults with facioscapulohumeral muscular dystrophy is currently recr…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD [Phase 2]
A Phase 2 study of apitegromab for facioscapulohumeral muscular dystrophy (FSHD) is listed as not yet recruiting.
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CLINICAL_TRIALS research · 2026-02-25Clinical Trial Update: Clenbuterol to Target DUX4 in FSHD [Phase 1]
A Phase 1 clinical trial is ongoing to test clenbuterol as a therapy targeting DUX4 in facioscapulohumeral muscular dystrophy (FSHD) and is …
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CLINICAL_TRIALS research · 2026-02-20Clinical Trial Update: BetterLife FSHD: A Patient-driven Health and Research Platform
The BetterLife FSHD trial platform, sponsored by the FSHD Society, is currently recruiting participants. It focuses on facioscapulohumeral m…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: New Clinical Outcome Measures to Remotely Evaluate Patients With FacioScapuloHumeral Muscular Dystrophy [NA]
This update describes the development of new remote clinical outcome measures for evaluating patients with facioscapulohumeral muscular dyst…
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CLINICAL_TRIALS research · 2026-02-06Clinical Trial Update: Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1 [Phase 1, Phase 2]
An ongoing Phase 1/2 clinical trial evaluating ARO-DUX4 in adults and adolescents with facioscapulohumeral muscular dystrophy type 1. The st…
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CLINICAL_TRIALS research · 2026-02-06Clinical Trial Update: Dietary Protein Requirements in Adults With Facioscapulohumeral Muscular Dystrophy [NA]
Clinical trial evaluating dietary protein requirements in adults with facioscapulohumeral muscular dystrophy is currently recruiting.
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD [Phase 3]
A phase 3 clinical trial evaluating Del-brax (AOC-1020) for facioscapulohumeral muscular dystrophy is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-08Clinical Trial Update: Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) [Phase 1]
Phase 1 clinical trial update on ULSC for facioscapulohumeral muscular dystrophy, highlighting safety assessments and preliminary efficacy s…
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CLINICAL_TRIALS research · 2025-12-03Clinical Trial Update: Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study [NA]
A prospective 24-month MRI study of facioscapulohumeral muscular dystrophy (FSHD) in France, sponsored by Centre Hospitalier Universitaire d…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Biomarker Development for Muscular Dystrophies
Massachusetts General Hospital sponsors a clinical trial update focused on developing biomarkers for muscular dystrophy. The study is curren…
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
A registry study update listing RECRUITING status for Myotonic Dystrophy type 1, Myotonic Dystrophy type 2, and Facioscapulohumeral muscular…
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CLINICAL_TRIALS research · 2025-09-25Clinical Trial Update: ADVANCED FSHD-COM: New Clinical Outcome Measures to Evaluate Non-ambulant FSHD Patients, a Pilot Study [NA]
This clinical trial update describes a pilot study to develop and evaluate new clinical outcome measures for non-ambulant patients with faci…
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Development and validation of the FBIndex, a tool to assess falls risk in patients with various neuromuscular disorders.
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Imaging and Gait Analysis in FSHD Patients
Clinical trial update indicating ongoing enrollment by invitation for an imaging and gait analysis study in patients with facioscapulohumera…
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CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology [NA]
This clinical trial update describes the assessment of gait analysis parameters and upper-limb evaluation in adults with various neurologica…
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CLINICAL_TRIALS research · 2025-07-09Clinical Trial Update: Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy
Update on a clinical study investigating MRI and MR spectroscopy biomarkers for facioscapulohumeral muscular dystrophy; the trial is current…
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CLINICAL_TRIALS research · 2025-04-27Clinical Trial Update: Personalized Training for People With Rare Neuromuscular Disorders [NA]
A clinical trial update announcing recruitment for personalized training programs for people with rare neuromuscular disorders. The study ta…
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CLINICAL_TRIALS research · 2025-04-24Clinical Trial Update: Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
A new clinical trial called MOVE FSHD is recruiting to validate motor outcome measures in facioscapulohumeral muscular dystrophy. The sponso…
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CLINICAL_TRIALS research · 2025-04-08Clinical Trial Update: Muscle MRI Outlining of Neuromuscular Diseases Using Artificial Intelligence
This clinical trial update describes a study using artificial intelligence to outline neuromuscular disease features on MRI. The conditions …
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Ten Year Follow-up in FSHD: the FOCUS 3 Study
This update covers the FOCUS 3 study for facioscapulohumeral muscular dystrophy and indicates that recruitment is ongoing for its ten-year f…