Publisher, "Facioscapulohumeral Muscular Dystrophy - Pipeline Insight, 2021," report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Facioscapulohumeral Muscular Dystrophy pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Facioscapulohumeral Muscular Dystrophy Understanding
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). FSHD may initially involve weakness of muscles of the face, shoulder girdle and arms. Facial weakness may result in limited movements of the lips, causing difficulties whistling, using a straw, or puckering the lips. Affected individuals may also develop a distinctive "mask-like" facial appearance. Upper facial weakness may also lead to an inability to completely close the eyes during sleep. FSHD may initially involve weakness of muscles of the face, shoulder girdle and arms. Facial weakness may result in limited movements of the lips, causing difficulties whistling, using a straw, or puckering the lips. Affected individuals may also develop a distinctive "mask-like" facial appearance. Upper facial weakness may also lead to an inability to completely close the eyes during sleep. FSHD may be diagnosed based upon a thorough clinical examination, identification of characteristic physical findings, a complete individual and family history, and genetic testing. In some affected individuals, laboratory studies may reveal elevated levels of a particular enzyme in the fluid portion of the blood (serum creatine kinase). Tests may also be conducted to record electrical activity in voluntary (skeletal) muscles at rest and during muscle contraction (electromyography [EMG]). Disease management may include orthopedic measures and physical therapy to help maintain muscle flexibility, counter atrophy and manage pain.
"Facioscapulohumeral Muscular Dystrophy - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Facioscapulohumeral Muscular Dystrophy pipeline landscape is provided which includes the disease overview and Facioscapulohumeral Muscular Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Facioscapulohumeral Muscular Dystrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Facioscapulohumeral Muscular Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Facioscapulohumeral Muscular Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve Facioscapulohumeral Muscular Dystrophy.
Facioscapulohumeral Muscular Dystrophy Emerging Drugs Chapters
This segment of the Facioscapulohumeral Muscular Dystrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Facioscapulohumeral Muscular Dystrophy Emerging Drugs
- Losmapimod: Fulcrum Therapeutics
The Company is evaluating the efficacy and safety of losmapimod in patients with FSHD in the randomized, double-blind, placebo-controlled phase IIb ReDUX4 trial. Fulcrum Therapeutics announces U.S. Food and Drug Administration grants Fast Track designation to losmapimod for the potential treatment of facioscapulohumeral muscular dystrophy.
- ATYR1940: aTyr Pharma, Inc.
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. 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), and Duchenne muscular dystrophy (DMD). It is currently being evaluated in Phase II stage of development for the treatment of Facioscapulohumeral Muscular Dystrophy.
Further product details are provided in the report..
Facioscapulohumeral Muscular Dystrophy: Therapeutic Assessment
This segment of the report provides insights about the different Facioscapulohumeral Muscular Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Facioscapulohumeral Muscular Dystrophy
There are approx. 8+ 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 II include, Fulcrum Therapeutics.
- Phases
Publisher report covers around 8+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Facioscapulohumeral Muscular Dystrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- Intravenous
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Facioscapulohumeral Muscular Dystrophy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Facioscapulohumeral Muscular Dystrophy therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Facioscapulohumeral Muscular Dystrophy drugs.
Facioscapulohumeral Muscular Dystrophy Report Insights
- Facioscapulohumeral Muscular Dystrophy Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugsx
Facioscapulohumeral Muscular Dystrophy Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Facioscapulohumeral Muscular Dystrophy drugs-
- How many Facioscapulohumeral Muscular Dystrophy drugs are developed by each company-
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Facioscapulohumeral Muscular Dystrophy-
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Facioscapulohumeral Muscular Dystrophy therapeutics-
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies-
- What are the clinical studies going on for Facioscapulohumeral Muscular Dystrophy and their status-
- What are the key designations that have been granted to the emerging drugs-
Table of Contents
Introduction
Executive Summary
Facioscapulohumeral Muscular Dystrophy: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Mid Stage Products (Phase II)
- Comparative Analysis
Losmapimod: Fulcrum Therapeutics
- Product Description
- Research and Development
- Product Development Activities
Drug profiles in the detailed report..
Early Stage Products (Phase I/II)
- Comparative Analysis
ATYR1940: aTyr Pharma, Inc.
- Product Description
- Research and Development
- Product Development Activities
Drug profiles in the detailed report..
Preclinical stage products
- Comparative Analysis
ARO DUX4: Arrowhead Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Drug profiles in the detailed report..
Inactive Products
- Comparative Analysis
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
List of Tables
Table 1 Total Products for Facioscapulohumeral Muscular Dystrophy
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
List of Figures
Figure 1 Total Products for Facioscapulohumeral Muscular Dystrophy
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
- aTyr Pharma, Inc.
- Fulcrum Therapeutics
- Avidity Biosciences
- Arrowhead Pharmaceuticals
- Dyne Therapeutics
- Genea Biocells
- Evotec SE/Facio Therapies
- miRecule
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