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Limb Girdle Muscular Dystrophy Clinical Trials Assessment 2024: FDA Approvals, Drugs, Clinical Trials, Therapeutic Analysis, and Companies by DelveInsight | Catabasis Pharmaceuticals, Sarepta Therapeutics, Strykagen, Sarepta Therapeutics, more
Limb Girdle Muscular Dystrophy Clinical Trials
"Limb Girdle Muscular Dystrophy Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Limb Girdle Muscular Dystrophy Market.
The Limb Girdle Muscular Dystrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
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Some of the key takeaways from the Limb Girdle Muscular Dystrophy Pipeline Report:
• Limb Girdle Muscular Dystrophy Companies across the globe are diligently working toward developing novel Limb Girdle Muscular Dystrophy treatment therapies with a considerable amount of success over the years. Limb Girdle Muscular Dystrophy Key players such as - Catabasis Pharmaceuticals, Sarepta Therapeutics, Strykagen, Sarepta Therapeutics, aTyr Pharma, ML Bio Solutions, and others, are developing therapies for the Limb Girdle Muscular Dystrophy treatment
• Limb Girdle Muscular Dystrophy Emerging therapies such as - CAT-1004, SRP-9005, Stryka 516s, MYO 102, SRP-9003, ATYR1940, BBP-418, and others are expected to have a significant impact on the Limb Girdle Muscular Dystrophy market in the coming years.
• ML Bio Solutions in February 2021, initiated an Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003). BBP-418 is being developed for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I).
• In January 2024, Sarepta Therapeutics has initiated the screening process for its Phase III EMERGENE clinical trial (Study SRP-9003-301) for the investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec) intended to treat limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), also known as beta sarcoglycanopathy. This multinational trial, conducted in an open-label format, aims to evaluate SRP-9003 in 15 participants.
• In August 2023, Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy firm fully owned and operated as a subsidiary of Bayer AG, disclosed today that the initial patient has received a dose in the Phase 1 / Phase 2 LION-CS101 clinical trial targeting patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). AB-1003 (also referred to as LION-101) is an innovative investigational therapy aimed at replacing the FKRP gene.
Limb Girdle Muscular Dystrophy Overview
Limb-girdle muscular dystrophy (LGMD) is a group of genetic disorders characterized by progressive muscle weakness and wasting, primarily affecting the shoulder and hip girdles. Onset can occur at any age, from childhood to adulthood, with the rate of progression varying widely among individuals.
Limb Girdle Muscular Dystrophy is caused by mutations in various genes responsible for muscle function, structure, and repair. These mutations lead to the deterioration of muscle fibers, resulting in the characteristic weakness and atrophy. The inheritance pattern can be autosomal dominant or recessive, depending on the specific type of LGMD.
Limb Girdle Muscular Dystrophy Symptoms typically include difficulty lifting objects, climbing stairs, and rising from a seated position. Over time, individuals may experience difficulty walking and may require mobility aids. Cardiac and respiratory muscles can also be affected in some types, leading to additional complications.
Limb Girdle Muscular Dystrophy Diagnosis involves a combination of clinical evaluation, family history, genetic testing, electromyography (EMG), and muscle biopsy. While there is no cure for Limb Girdle Muscular Dystrophy, management focuses on improving quality of life through physical therapy, orthopedic interventions, and respiratory support when needed. Research is ongoing to develop gene therapies and other treatments to slow disease progression and improve outcomes for those affected by Limb Girdle Muscular Dystrophy.
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Limb Girdle Muscular Dystrophy Pipeline Therapeutics Assessment
• Limb Girdle Muscular Dystrophy Assessment by Product Type
• Limb Girdle Muscular Dystrophy By Stage and Product Type
• Limb Girdle Muscular Dystrophy Assessment by Route of Administration
• Limb Girdle Muscular Dystrophy By Stage and Route of Administration
• Limb Girdle Muscular Dystrophy Assessment by Molecule Type
• Limb Girdle Muscular Dystrophy by Stage and Molecule Type
DelveInsight's Limb Girdle Muscular Dystrophy Report covers around 25+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration
Download Sample PDF Report to know more about Limb Girdle Muscular Dystrophy drugs and therapies- https://www.delveinsight.com/sample-request/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr
Emerging Limb Girdle Muscular Dystrophy Drugs Under Different Phases of Clinical Development Include:
• CAT-1004: Catabasis Pharmaceuticals
• SRP-9005: Sarepta Therapeutics
• Stryka 516s: Strykagen
• MYO 102: Sarepta Therapeutics
• SRP-9003: Sarepta Therapeutics
• ATYR1940: aTyr Pharma
• BBP-418: ML Bio Solutions
Limb Girdle Muscular Dystrophy Pipeline Analysis:
The Limb Girdle Muscular Dystrophy pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Limb Girdle Muscular Dystrophy treatment with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Limb Girdle Muscular Dystrophy Treatment.
• Limb Girdle Muscular Dystrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Limb Girdle Muscular Dystrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Limb Girdle Muscular Dystrophy market.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
Further Limb Girdle Muscular Dystrophy product details are provided in the report. Download the Limb Girdle Muscular Dystrophy pipeline report to learn more about the emerging Limb Girdle Muscular Dystrophy therapies at:
https://www.delveinsight.com/sample-request/limb-girdle-muscular-dystrophy-lgmd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr
Limb Girdle Muscular Dystrophy Pipeline Market Drivers
• Increasing awareness about available treatment options
• A better understanding of disease pathogenesis
Limb Girdle Muscular Dystrophy Pipeline Market Barriers
• No cure exists for any form of Limb Girdle Muscular Dystrophy
• Lack of proper Genetic Counselling, which is to be given to individuals and their families.
Scope of Limb Girdle Muscular Dystrophy Pipeline Drug Insight
• Coverage: Global
• Key Limb Girdle Muscular Dystrophy Companies: Catabasis Pharmaceuticals, Sarepta Therapeutics, Strykagen, Sarepta Therapeutics, aTyr Pharma, ML Bio Solutions, and others
• Key Limb Girdle Muscular Dystrophy Therapies: CAT-1004, SRP-9005, Stryka 516s, MYO 102, SRP-9003, ATYR1940, BBP-418, and others
• Limb Girdle Muscular Dystrophy Therapeutic Assessment: Limb Girdle Muscular Dystrophy current marketed and Limb Girdle Muscular Dystrophy emerging therapies
• Limb Girdle Muscular Dystrophy Market Dynamics: Limb Girdle Muscular Dystrophy market drivers and Limb Girdle Muscular Dystrophy market barriers
Request for Sample PDF Report for Limb Girdle Muscular Dystrophy Pipeline Assessment and clinical trials-
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Table of Contents
1 Limb Girdle Muscular Dystrophy Report Introduction
2 Limb Girdle Muscular Dystrophy Executive Summary
3 Limb Girdle Muscular Dystrophy Overview
4 Limb Girdle Muscular Dystrophy- Analytical Perspective In-depth Commercial Assessment
5 Limb Girdle Muscular Dystrophy Pipeline Therapeutics
6 Limb Girdle Muscular Dystrophy Late Stage Products (Phase II/III)
7 Limb Girdle Muscular Dystrophy Mid Stage Products (Phase II)
8 Limb Girdle Muscular Dystrophy Early Stage Products (Phase I)
9 Limb Girdle Muscular Dystrophy Preclinical Stage Products
10 Limb Girdle Muscular Dystrophy Therapeutics Assessment
11 Limb Girdle Muscular Dystrophy Inactive Products
12 Company-University Collaborations (Licensing/Partnering) Analysis
13 Limb Girdle Muscular Dystrophy Key Companies
14 Limb Girdle Muscular Dystrophy Key Products
15 Limb Girdle Muscular Dystrophy Unmet Needs
16 Limb Girdle Muscular Dystrophy Market Drivers and Barriers
17 Limb Girdle Muscular Dystrophy Future Perspectives and Conclusion
18 Limb Girdle Muscular Dystrophy Analyst Views
19 Appendix
20 About DelveInsight
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