Limb Girdle Muscular Dystrophy - Pipeline Insight, 2024

This “Limb Girdle Muscular Dystrophy - Pipeline Insight, 2024,” report provides comprehensive insights about 20+ companies and 25+ pipeline drugs in Limb Girdle 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

Limb Girdle Muscular Dystrophy Understanding

Limb Girdle Muscular Dystrophy: Overview

Limb-girdle muscular dystrophies (LGMD) are a group of rare progressive genetic disorders that are characterized by wasting (atrophy) and weakness of the voluntary muscles of the hip and shoulder areas (limb-girdle area). Muscle weakness and atrophy are progressive and may spread to affect other muscles of the body. Many different subtypes have been identified based upon abnormal changes (mutations) of certain genes. The age at onset, severity, and progression of symptoms of these subtypes may vary greatly from case to case, even among individuals in the same family. Some individuals may have a mild, slowly progressive form of the disorders; other may have a rapidly progressive form of the disorder that causes severe disability. The term limb-girdle muscular dystrophies is a general term that encompasses several disorders. These disorders can now be distinguished by genetic and protein analysis. The various forms of LGMD may be inherited as autosomal dominant or recessive traits. Autosomal dominant LGMD is known as LGMD1 and there are currently recognized eight subtypes (LGMD1A-1H). Autosomal recessive LGMD is known as LGMD2 and has 17 subtypes (LGMDA-Q).

Limb Girdle Muscular Dystrophy - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Limb Girdle Muscular Dystrophy pipeline landscape is provided which includes the disease overview and Limb Girdle Muscular Dystrophy treatment guidelines. The assessment part of the report embraces, in depth Limb Girdle 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, Limb Girdle Muscular Dystrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Limb Girdle Muscular Dystrophy.
• In the coming years, the Limb Girdle Muscular Dystrophy market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

The companies and academics that are working to assess challenges and seek opportunities that could influence Limb Girdle Muscular Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• A detailed portfolio of major pharma players who are involved in fueling the Limb Girdle Muscular Dystrophy treatment market. Several potential therapies for Limb Girdle Muscular Dystrophy are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Limb Girdle Muscular Dystrophy market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Limb Girdle Muscular Dystrophy) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Limb Girdle Muscular Dystrophy Emerging Drugs Chapters

This segment of the Limb Girdle 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.

Limb Girdle Muscular Dystrophy Emerging Drugs

BBP-418: ML Bio Solutions BBP-418 is a sugar that the body produces naturally and supplements the substrate that is incorporated into alpha dystroglycan to allow it to function normally. Published studies have validated the role of BBP-418 as a limiting constituent to alpha dystroglycan’s shock absorber function. ML Bio Solutions is investigating BBP-418 as a potential therapy for treating patients affected by mutations to FKRP. Currently, it is in Phase II stage of clinical trial evaluation to treat Limb girdle musculardystrophies.

LION-101: Asklepios Bio Pharmaceutical LION-101 is a novel recombinant adeno-associated virus (r AAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Currently, it is being evaluated in Phase I/II stage of clinical trial evaluation to treat Limbgirdlemusculardystrophies.

Limb Girdle Muscular Dystrophy: Therapeutic Assessment

This segment of the report provides insights about the different Limb Girdle Muscular Dystrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Limb Girdle Muscular Dystrophy

There are approx. 20+ key companies which are developing the therapies for Limb Girdle Muscular Dystrophy. The companies which have their Limb Girdle Muscular Dystrophy drug candidates in the most advanced stage, i.e. phase II include, ML Bio Solutions.

Phases

This 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) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Limb Girdle 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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Limb Girdle Muscular Dystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Limb Girdle 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 Limb Girdle Muscular Dystrophy drugs.

Limb Girdle Muscular Dystrophy Report Insights

• Limb Girdle Muscular Dystrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Limb Girdle 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 Limb Girdle Muscular Dystrophy drugs?
• How many Limb Girdle 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 Limb Girdle Muscular Dystrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Limb Girdle 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 Limb Girdle Muscular Dystrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• ML Bio Solutions
• Asklepios BioPharmaceutical
• Sarepta Therapeutics, Inc.
• Atamyo Therapeutics
• Edgewise Therapeutics
• Vita Therapeutics
• BridgeBio Pharma
• Hansa Biopharma

Key Products

• BBP-418
• LION-101
• SRP-9003
• GNT0006
• SRP-9004
• EDG-5506
• VTA-100

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