Facioscapulohumeral Muscular Dystrophy Market: Pharma Pipeline Fuels Rapid Expansion - DelveInsight | Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics

The Key Facioscapulohumeral Muscular Dystrophy Companies in the market include - Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics, and others.
DelveInsight's "Facioscapulohumeral Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast-2034 report offers an in-depth understanding of the Facioscapulohumeral Muscular Dystrophy, historical and forecasted epidemiology as well as the Facioscapulohumeral Muscular Dystrophy market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Facioscapulohumeral Muscular Dystrophy Market Report:

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The Facioscapulohumeral Muscular Dystrophy market size is anticipated to grow with a significant CAGR during the study period (2020-2034).

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In March 2026, Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a leader in precision genetic therapies for rare diseases, announced initial clinical findings from two of its siRNA programs targeting neuromuscular disorders. Early data from Phase 1/2 dose-escalation studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1) showed dose-related muscle exposure, early biomarker activity, and good tolerability, supporting confidence in the potential advantages of its v6 integrin-targeted delivery platform. Additionally, the company reported proof-of-concept results indicating that a single dose of both SRP-1001 and SRP-1003 can reduce, or knock down, the targeted protein or mRNA. Across both studies, most adverse events were mild to moderate and did not increase with higher doses.

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In June 2025, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company advancing a novel class of RNA therapeutics known as Antibody Oligonucleotide Conjugates (AOCs Trademark ), announced that it will deliver two oral presentations and one poster at the 32nd Annual FSHD Society International Research Congress, scheduled for June 12-13, 2025, in Amsterdam, Netherlands. Earlier this week, the company also reported alignment with the FDA on both accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral muscular dystrophy (FSHD).

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In April 2025, Epicrispr Biotechnologies, a biotechnology firm focused on developing curative therapies, has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for its candidate EPI-321. This innovative treatment represents the first epigenetic-based approach targeting facioscapulohumeral muscular dystrophy (FSHD), a genetic neuromuscular condition.

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In March 2025, Epicrispr Biotechnologies has secured $68 million in the initial closing of its Series B funding round to initiate clinical trials for its disease-modifying therapy, EPI-321, targeting facioscapulohumeral muscular dystrophy (FSHD). The funds will also support the clinical advancement of this treatment for the genetic neuromuscular disorder, along with the continued development of the company's broader therapeutic pipeline.

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In March 2025, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company advancing a new class of RNA-based medicines known as Antibody Oligonucleotide Conjugates (AOCs Trademark ), has announced the completion of enrollment for the biomarker cohort in its Phase 1/2 FORTITUDE Trademark clinical trial. The study is evaluating delpacibart braxlosiran (del-brax) in individuals with facioscapulohumeral muscular dystrophy (FSHD), with a total of 51 participants enrolled in this cohort.

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In October 2024, Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company advancing RNA therapeutics through Antibody Oligonucleotide Conjugates (AOCs Trademark ), has launched a biomarker cohort in the Phase 1/2 FORTITUDE Trademark trial of delpacibart braxlosiran (del-brax/AOC 1020) for individuals with facioscapulohumeral muscular dystrophy (FSHD). The company is exploring a potential accelerated approval pathway for del-brax, with plans to complete biomarker cohort enrollment by mid-2025. Additionally, a functional cohort is set to begin in the first half of 2025, and enrollment for the FORTITUDE Open-label Extension (OLE) study is ongoing.

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In July 2024, The University of Kansas (KU) Medical Center in the US has shared initial findings from the Phase I/II trial of Avidity Biosciences' drug, del-brax (delpacibart braxlosiran), aimed at treating facioscapulohumeral muscular dystrophy (FSHD). As the trial's study site, KU Medical Center reported early results suggesting that del-brax may substantially reduce the expression of the DUX4 gene.

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In 2023, the total prevalent cases of FSHD in the 7MM were approximately 79,000, with an expected increase by 2034.

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In 2023, the United States accounted for roughly 40% of the total cases in the 7MM, with these numbers expected to rise by 2034.

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According to DelveInsight's analysts, there were approximately 6,000 cases of FSHD1 and about 300 cases of FSHD2 in Japan in 2023. These numbers are expected to decrease during the forecast period (2024-2034).

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According to DelveInsight's consultant estimates, the majority of cases among the seven major markets are attributed to FSHD1. In 2023, approximately 95% of the prevalent cases were FSHD1, as compared to FSHD2.

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During the forecast period (2024-2034), pipeline candidates like losmapimod (Fulcrum Therapeutics) and RO7204239/GYM-329/RG-6237 (Hoffmann-La Roche) are anticipated to contribute to the growth of the FSHD market size.

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In 2023, the total number of treated cases of FSHD in the 7MM was approximately 34,000, with an anticipated increase by 2034.

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In 2023, the highest number of age-specific cases of FSHD in the 7MM was observed in patients aged 50 years and above, totaling approximately 23,000. These numbers are projected to rise in the forecast period.

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Key Facioscapulohumeral Muscular Dystrophy Companies: Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics, and others

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Key Facioscapulohumeral Muscular Dystrophy Therapies: DYNE-301, MC-DX4, AOC 1020, ATYR 1940, Losmapimod, and others

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The Facioscapulohumeral Muscular Dystrophy market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Facioscapulohumeral Muscular Dystrophy pipeline products will significantly revolutionize the Facioscapulohumeral Muscular Dystrophy market dynamics.

Facioscapulohumeral Muscular Dystrophy Overview

Facioscapulohumeral Muscular Dystrophy (FSHD) is a genetic muscle disorder characterized by progressive muscle weakness and wasting, primarily affecting the muscles of the face (facio-), shoulder blades (scapulo-), and upper arms (humeral). It is one of the most common forms of muscular dystrophy.

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Facioscapulohumeral Muscular Dystrophy Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Facioscapulohumeral Muscular Dystrophy Epidemiology Segmentation:

The Facioscapulohumeral Muscular Dystrophy market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Facioscapulohumeral Muscular Dystrophy

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Prevalent Cases of Facioscapulohumeral Muscular Dystrophy by severity

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Gender-specific Prevalence of Facioscapulohumeral Muscular Dystrophy

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Diagnosed Cases of Episodic and Chronic Facioscapulohumeral Muscular Dystrophy

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Facioscapulohumeral Muscular Dystrophy Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Facioscapulohumeral Muscular Dystrophy market or expected to get launched during the study period. The analysis covers Facioscapulohumeral Muscular Dystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Facioscapulohumeral Muscular Dystrophy Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Facioscapulohumeral Muscular Dystrophy Therapies and Key Companies

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DYNE-301: Dyne Therapeutics

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MC-DX4: miRecule

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AOC 1020: Avidity Biosciences

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ATYR 1940: aTyr Pharma

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Losmapimod: Fulcrum Therapeutics

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Facioscapulohumeral Muscular Dystrophy Market Strengths

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Government registries aim to facilitate and accelerate academic and clinical research in FSHD, leading to the increase in knowledge and awareness.

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Currently, there are no approved therapies to treat FSHD, so there will be a high chance of reimbursement for the upcoming therapies, as the cost of the treatment will be very high, and this will be first mover advantage for the government to reimburse the drugs

Facioscapulohumeral Muscular Dystrophy Market Opportunities

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Limitations in current drug therapies may lead to the need for more and more reliable treatment, which could also fuel demand growth

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Developing novel therapeutics and individualized medical care based on biomarkers will present profitable commercial prospects

Scope of the Facioscapulohumeral Muscular Dystrophy Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Facioscapulohumeral Muscular Dystrophy Companies: Dyne Therapeutics, miRecule, Avidity Biosciences, aTyr Pharma, Fulcrum Therapeutics, and others

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Key Facioscapulohumeral Muscular Dystrophy Therapies: DYNE-301, MC-DX4, AOC 1020, ATYR 1940, Losmapimod, and others

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Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment: Facioscapulohumeral Muscular Dystrophy current marketed and Facioscapulohumeral Muscular Dystrophy emerging therapies

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Facioscapulohumeral Muscular Dystrophy Market Dynamics: Facioscapulohumeral Muscular Dystrophy market drivers and Facioscapulohumeral Muscular Dystrophy market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Facioscapulohumeral Muscular Dystrophy Unmet Needs, KOL's views, Analyst's views, Facioscapulohumeral Muscular Dystrophy Market Access and Reimbursement

To know more about Facioscapulohumeral Muscular Dystrophy companies working in the treatment market, visit @ Facioscapulohumeral Muscular Dystrophy Clinical Trials and Treatment [https://www.delveinsight.com/sample-request/facioscapulohumeral-muscular-dystrophy-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Facioscapulohumeral Muscular Dystrophy Market Report Introduction

2. Executive Summary for Facioscapulohumeral Muscular Dystrophy

3. SWOT analysis of Facioscapulohumeral Muscular Dystrophy

4. Facioscapulohumeral Muscular Dystrophy Patient Share (%) Overview at a Glance

5. Facioscapulohumeral Muscular Dystrophy Market Overview at a Glance

6. Facioscapulohumeral Muscular Dystrophy Disease Background and Overview

7. Facioscapulohumeral Muscular Dystrophy Epidemiology and Patient Population

8. Country-Specific Patient Population of Facioscapulohumeral Muscular Dystrophy

9. Facioscapulohumeral Muscular Dystrophy Current Treatment and Medical Practices

10. Facioscapulohumeral Muscular Dystrophy Unmet Needs

11. Facioscapulohumeral Muscular Dystrophy Emerging Therapies

12. Facioscapulohumeral Muscular Dystrophy Market Outlook

13. Country-Wise Facioscapulohumeral Muscular Dystrophy Market Analysis (2020-2034)

14. Facioscapulohumeral Muscular Dystrophy Market Access and Reimbursement of Therapies

15. Facioscapulohumeral Muscular Dystrophy Market Drivers

16. Facioscapulohumeral Muscular Dystrophy Market Barriers

17. Facioscapulohumeral Muscular Dystrophy Appendix

18. Facioscapulohumeral Muscular Dystrophy Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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