Facioscapulohumeral Muscular Dystrophy Pipeline 2024 | Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc.

DelveInsight's, "Facioscapulohumeral Muscular Dystrophy Pipeline Insight 2024" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Facioscapulohumeral Muscular Dystrophy pipeline landscape. It covers the Facioscapulohumeral Muscular Dystrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Facioscapulohumeral Muscular Dystrophy 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.

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Key Takeaways from the Facioscapulohumeral Muscular Dystrophy Pipeline Report

* July 2024:- Hoffmann-La Roche - A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy.
* DelveInsight's Facioscapulohumeral Muscular Dystrophy pipeline report depicts a robust space with 10+ active players working to develop 10+ pipeline therapies for Facioscapulohumeral Muscular Dystrophy treatment.
* The leading Facioscapulohumeral Muscular Dystrophy Companies such as Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc. , and others.
* Promising Facioscapulohumeral Muscular Dystrophy Therapies such as Losmapimod, RO7204239, AOC 1020 , and others.

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Facioscapulohumeral Muscular Dystrophy Emerging Drugs Profile

* Losmapimod: Fulcrum Therapeutics

Losmapimod is an investigational, selective p38/ mitogen activated protein kinase (MAPK) inhibitor. Fulcrum exclusively in-licensed losmapimod from GSK following Fulcrum's discovery of the role of p38/ 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.

* DYNE-301: Dyne Therapeutics

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. Currently the product is in preclinical stage of development for the disease.

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Facioscapulohumeral Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration

* Intra-articular
* Intraocular
* Intrathecal
* Intravenous
* Ophthalmic
* Oral
* Parenteral
* Subcutaneous
* Topical
* Transdermal

Facioscapulohumeral Muscular Dystrophy Products have been categorized under various Molecule types such as

* Oligonucleotide
* Peptide
* Small molecule

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Scope of the Facioscapulohumeral Muscular Dystrophy Pipeline Report

* Coverage- Global
* Facioscapulohumeral Muscular Dystrophy Companies- Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc., and others.
* Facioscapulohumeral Muscular Dystrophy Therapies- Losmapimod, RO7204239, AOC 1020, and others.
* Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Facioscapulohumeral Muscular Dystrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* Introduction
* Executive Summary
* Facioscapulohumeral Muscular Dystrophy: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Facioscapulohumeral Muscular Dystrophy- DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Losmapimod: Fulcrum Therapeutics
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* DYNE-301: Dyne Therapeutics
* Drug profiles in the detailed report.....
* Inactive Products
* Facioscapulohumeral Muscular Dystrophy Key Companies
* Facioscapulohumeral Muscular Dystrophy Key Products
* Facioscapulohumeral Muscular Dystrophy- Unmet Needs
* Facioscapulohumeral Muscular Dystrophy- Market Drivers and Barriers
* Facioscapulohumeral Muscular Dystrophy- Future Perspectives and Conclusion
* Facioscapulohumeral Muscular Dystrophy Analyst Views
* Facioscapulohumeral Muscular Dystrophy Key Companies
* Appendix

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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