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Facioscapulohumeral Muscular Dystrophy Pipeline & Clinical Trial Report | 12+ Companies and 12+ Drugs

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Facioscapulohumeral Muscular Dystrophy Pipeline & Clinical Trial Report | 12+ Companies and 12+ Drugs

January 12
20:24 2023
Facioscapulohumeral Muscular Dystrophy Pipeline & Clinical Trial Report | 12+ Companies and 12+ Drugs

DelveInsight’s, “Facioscapulohumeral Muscular Dystrophy Pipeline Insight, 2023,” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Facioscapulohumeral Muscular Dystrophy pipeline landscape. It covers the Facioscapulohumeral Muscular Dystrophy pipeline drug profiles, including Facioscapulohumeral Muscular Dystrophy clinical trials and nonclinical stage products. It also covers the Facioscapulohumeral Muscular Dystrophy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive Facioscapulohumeral Muscular Dystrophy pipeline products in this space.

 

Key takeaways from the Facioscapulohumeral Muscular Dystrophy Pipeline Report

 

  • DelveInsight’s Facioscapulohumeral Muscular Dystrophy pipeline report depicts a robust space with 12+ active players working to develop 12+ pipeline therapies for Facioscapulohumeral Muscular Dystrophy treatment.

 

  • The leading Facioscapulohumeral Muscular Dystrophy Companies include Fulcrum Therapeutics, aTyr Pharma, Myocea, Dyne Therapeutics, Avidity Biosciences, Arrowhead Pharmaceuticals, Healx, and others.

 

  • Promising Facioscapulohumeral Muscular Dystrophy Pipeline Therapies include Losmapimod, ATYR1940, MYO-029, and others.

 

  • The Facioscapulohumeral Muscular Dystrophy Companies and academics are working to assess challenges and seek opportunities that could influence Facioscapulohumeral Muscular Dystrophy R&D. The Facioscapulohumeral Muscular Dystrophy pipeline therapies under development are focused on novel approaches to treat Facioscapulohumeral Muscular Dystrophy.

 

Explore more about the latest breakthroughs of Facioscapulohumeral Muscular Dystrophy Treatment Landscape @ Facioscapulohumeral Muscular Dystrophy Pipeline Outlook

 

Facioscapulohumeral Muscular Dystrophy Overview

Facioscapulohumeral muscular dystrophy (FSHD) is a disorder characterized by muscle weakness and wasting (atrophy). The disorder gets its name from muscles that are affected in the face (facio), around the shoulder blades (scapulo), and in the upper arms (humeral). Hamstring and trunk muscles are affected -early on but are less well recognized. Other arm and leg muscles are frequently eventually affected in the course of the disease. Symptoms usually appear before age 20, but can begin in infancy or later in adulthood. Severity of the condition varies widely and some people with the disease allele remain asymptomatic. FSHD is most typically characterized by relatively slow disease progression. Specific symptoms and findings may also vary in range and severity, including among affected members of the same family. Life expectancy is not shortened. FSHD is usually inherited as an autosomal dominant genetic condition.

 

Recent Developmental Activities in the Facioscapulohumeral Muscular Dystrophy Treatment Landscape

 

  • Losmapimod is an investigational, selective p38α/β mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum’s discovery of the role of p38α/β inhibitors in the reduction of DUX4 expression and an extensive review of known compounds. Results reported from the ReDUX4 trial demonstrated slowed disease progression and improved function, including positive impacts on upper extremity strength, supporting losmapimod’s potential to be a transformative therapy for the treatment of FSHD. Although losmapimod had never previously been explored in muscular dystrophies, it had been evaluated in more than 3,600 subjects in clinical trials across multiple other indications, with no safety signals attributed to losmapimod. Losmapimod has been granted FDA Fast Track designation and Orphan Drug Designation for the treatment of FSHD. Fulcrum Therapeutics intends to submit regulatory application to the US FDA and EMA in Facioscapulohumeral muscular dystrophy. The drug is currently in Phase III stage of development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

 

  • ATYR1940 is based on a protein naturally secreted from muscle (Resokine) that may act to influence T-cell activation at the tissue level to promote healthier muscle. ATYR1940 as a potential first-in-class intravenous protein therapeutic candidate for the treatment of rare myopathies with an immune component. There is potential that ATYR1940 may translate into an innovative therapeutic for rare genetic myopathies with an immune component, including limb-girdle muscular dystrophy (LGMD), facioscapulohumeral muscular dystrophy (FSHD). Currently the product is in Phase I/II stage of development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

 

  • DYNE-301 is a DUX4L1 protein expression stimulant, which consists of company’s proprietary Fab conjugated with our linker to an ASO that is designed to address the genetic basis of FSHD by reducing DUX4 expression in muscle tissue. Proof-of-concept data showing that DYNE-301 reduced expression of key DUX4 biomarkers in FSHD patient myotubes, a type of muscle cell. Company anticipate submitting an IND for DYNE-301 in the second half of 2022. Currently the product is in preclinical stage of development for the treatment of Facioscapulohumeral Muscular Dystrophy.

 

Learn more about the Facioscapulohumeral Muscular Dystrophy Emerging Therapies @ Facioscapulohumeral Muscular Dystrophy Ongoing Clinical Trials Analysis

 

Facioscapulohumeral Muscular Dystrophy Emerging Drugs Profile

  • Losmapimod: Fulcrum Therapeutics
  • ATYR1940: aTyr Pharma
  • DYNE-301: Dyne Therapeutics

 

Facioscapulohumeral Muscular Dystrophy Therapeutics Assessment

There are approx. 12+ key companies which are developing the therapies for Facioscapulohumeral Muscular Dystrophy. The companies which have their Facioscapulohumeral Muscular Dystrophy drug candidates in the most advanced stage, i.e. phase III include, Fulcrum Therapeutics.

 

Dive deep into rich insights for new drugs for Facioscapulohumeral Muscular Dystrophy Companies and drugs; visit @ Facioscapulohumeral Muscular Dystrophy Treatment Landscape

 

Scope of the Facioscapulohumeral Muscular Dystrophy Pipeline Report

  • Coverage- Global
  • CompaniesFulcrum Therapeutics, aTyr Pharma, Myocea, Dyne Therapeutics, Avidity Biosciences, Arrowhead Pharmaceuticals, Healx, and others
  • Pipeline Therapies- Losmapimod, ATYR1940, MYO-029, and others
  • Facioscapulohumeral Muscular Dystrophy Segmentation: Phases, Molecule Type, Mechanism of Action, Route of Administration, Product Type

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Facioscapulohumeral Muscular Dystrophy: Overview
  4. Facioscapulohumeral Muscular Dystrophy Pipeline Therapeutics
  5. Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment
  6. Facioscapulohumeral Muscular Dystrophy– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. Losmapimod: Fulcrum Therapeutics
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase I/II)
  11. ATYR1940: aTyr Pharma
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I)
  14. Drug name : Company name
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. DYNE-301: Dyne Therapeutics
  18. Drug profiles in the detailed report…..
  19. Inactive Facioscapulohumeral Muscular Dystrophy Products
  20. Facioscapulohumeral Muscular Dystrophy Key Companies
  21. Facioscapulohumeral Muscular Dystrophy Key Products
  22. Facioscapulohumeral Muscular Dystrophy Unmet Needs
  23. Facioscapulohumeral Muscular Dystrophy Market Drivers
  24. Facioscapulohumeral Muscular Dystrophy Market Barriers
  25. Facioscapulohumeral Muscular Dystrophy Future Perspectives and Conclusion
  26. Facioscapulohumeral Muscular Dystrophy Analyst Views
  27. Facioscapulohumeral Muscular Dystrophy Key Companies
  28. Appendix

 

For further information on the Facioscapulohumeral Muscular Dystrophy Pipeline therapeutics, reach out to Facioscapulohumeral Muscular Dystrophy Market Drivers and Barriers

 

About Us

DelveInsight is a Business Consulting and Market research company, providing expert business solutions for the healthcare domain and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.

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