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Report Overview
Limb girdle muscular dystrophy (LGMD) is a rare genetic neuromuscular disorder marked by progressive weakness in the hip and shoulder muscles, leading to mobility limitations. According to Expert Market Research, global prevalence is estimated between about 1 and 9 cases per 100,000 people, underscoring its rarity. The limb girdle muscular dystrophy drug pipeline is expanding with gene therapies, exon‑skipping, and AAV‑based approaches targeting specific genetic defects, while supportive care remains standard. Growing focus on precision medicine, enhanced diagnostics, and increased R&D investment is driving innovation and anticipated market growth in the coming years as unmet needs continue to shape therapeutic strategies.
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Major companies involved in the limb girdle muscular dystrophy pipeline analysis include Sarepta Therapeutics, Inc., AskBio Inc., and others.
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Leading drugs currently in the pipeline include SRP-9003, AB-1003, ATA-100, and others.
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The pipeline is poised for growth with increasing gene therapy and exon-skipping candidates, rising clinical trial activity, and expanding collaborations among biotech firms, driving innovation and potential treatment options in the coming years.
Report Coverage
The Limb Girdle Muscular Dystrophy Pipeline Analysis Report by Expert Market Research gives comprehensive insights into limb girdle muscular dystrophy therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for limb girdle muscular dystrophy. The limb girdle muscular dystrophy report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The limb girdle muscular dystrophy pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with limb girdle muscular dystrophy treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to limb girdle muscular dystrophy.
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Limb Girdle Muscular Dystrophy Pipeline Outlook
Limb-girdle muscular dystrophy (LGMD) is a rare genetic disorder causing progressive weakness and wasting of proximal muscles, primarily in the shoulders, upper arms, pelvic area, and thighs. It occurs due to mutations in specific genes, such as FKRP in LGMD2I/R9, disrupting normal muscle function and leading to gradual disability over time.
Limb-girdle muscular dystrophy treatment focuses on managing symptoms through physical therapy, assistive devices, and supportive care, as no approved therapy currently halts or reverses disease progression. In February 2023, AskBio’s EU subsidiary BrainVectis received European Commission orphan drug designation for AB-1003, a novel AAV-based gene therapy in development for LGMD2I/R9, targeting safety, tolerability, and efficacy through a one-time intravenous infusion in affected adults.
Limb Girdle Muscular Dystrophy Epidemiology
The pipeline is gaining focus due to the significant unmet medical need across its subtypes. According to Sarepta, the approximate global prevalence of all LGMDs is 1.63 per 100,000 people. As per Matthew P. Wicklund et al., 2025, around 20,000 individuals currently live with LGMD in the United States, with many subtypes considered ultra-rare, affecting only a few hundred to a few thousand patients per subtype. Additionally, a study by Xuelian Hong et al., 2024, in the United Kingdom found that 2 of 85 patients with LGMD R1/2A experienced atrial fibrillation and left ventricular dysfunction. Collectively, these data highlight the urgent need for effective therapies.
Limb Girdle Muscular Dystrophy – Pipeline Therapeutic Assessment
This section of the report covers the analysis of limb girdle muscular dystrophy drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
By Drug Class
The limb girdle muscular dystrophy pipeline analysis report covers 50+ drug analyses based on drug classes:
- Small Molecules
- Monoclonal Antibodies
- Peptides
- Proteins
- Vaccines
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration:
- Oral
- Parenteral
- Others
Limb Girdle Muscular Dystrophy Pipeline Assessment Segmentation, By Phases
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase I, with 44%, covers a major share of the total limb girdle muscular dystrophy clinical trials. It is followed by phase II at 33% and phase III at 22%. This robust distribution in clinical phases highlights advancing therapeutic prospects, potentially accelerating innovation and strengthening future treatment options in the market.
Limb Girdle Muscular Dystrophy Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the limb girdle muscular dystrophy pipeline analysis include small molecules, monoclonal antibodies, peptides, proteins, and vaccines. The limb girdle muscular dystrophy report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for limb girdle muscular dystrophy. Gene-targeted therapies are emerging as potential treatments for limb-girdle muscular dystrophy. For instance, BBP-418, an oral investigational therapy by BridgeBio Pharma, is under evaluation in a Phase 3 registrational study for LGMD2I/R9. BBP-418 aims to restore glycosylation of alpha-dystroglycan, improving muscle stability, and may qualify for Accelerated Approval in the United States based on interim biomarker analysis.
Limb Girdle Muscular Dystrophy Clinical Trials – Key Players
The EMR report for the limb girdle muscular dystrophy pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed limb girdle muscular dystrophy therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in limb girdle muscular dystrophy clinical trials:
- Sarepta Therapeutics, Inc.
- AskBio Inc.
- Atamyo Therapeutics
- ML Bio Solutions, Inc.
- aTyr Pharma, Inc.
- Pfizer
- Endo Pharmaceuticals
- Vernalis (R&D) Ltd.
- Celgene
Limb Girdle Muscular Dystrophy – Emerging Drugs Profile
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for limb girdle muscular dystrophy. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of limb girdle muscular dystrophy drug candidates.
Biological: SRP-9003
SRP-9003 (Badridistrogene Xeboparvovec) is an investigational systemic gene therapy sponsored by Sarepta Therapeutics, Inc., designed to treat limb girdle muscular dystrophy 2E/R4 (LGMD2E/R4). This Phase 3 study is examining the safety and efficacy of a single systemic dose of SRP-9003 in both ambulatory and non-ambulatory participants. The therapy employs an AAVrh74 viral vector to deliver a functional beta-sarcoglycan (β-SG) gene to skeletal, diaphragm, and cardiac muscles, with the MHCK7 promoter enhancing cardiac expression. Administered intravenously, SRP-9003 is working to restore β-SG protein production, aiming to improve muscle function and reduce cardiac and pulmonary complications associated with LGMD.
Genetic: AB-1003
AB-1003 is an investigational AAV-based gene therapy being developed by AskBio Inc. It is designed to restore FKRP enzyme activity in muscle cells for patients with limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This Phase 1 study is evaluating the safety and tolerability of a single intravenous infusion of AB-1003 in sequential, dose-level cohorts of adult participants. The study examines potential adverse effects, immune responses, and preliminary indications of therapeutic activity. AB-1003 works by delivering a functional FKRP gene directly to muscle tissue, aiming to improve muscle function and slow disease progression over time.
Drug: Biological: ATA-100
ATA-100 is an intravenous gene therapy sponsored by Atamyo Therapeutics, designed to treat FKRP-related limb-girdle muscular dystrophy (LGMDR9). This Phase 1 open-label, dose-escalation study assesses the safety, tolerability, pharmacodynamics, and immunogenicity of ATA-100, an AAV9-based vector carrying a functional FKRP transgene. The therapy works by delivering a normal copy of the FKRP gene to restore protein production, potentially improving muscle function. Two sequential dose cohorts are being enrolled, with patients followed for up to five years post-administration. ATA-100 has received Orphan Drug, Fast Track, and Rare Pediatric designations in the US and EU.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
Key Questions Answered in the Limb Girdle Muscular Dystrophy Pipeline Insight Report
- Which companies/institutions are leading the limb girdle muscular dystrophy drug development?
- Which company is leading the limb girdle muscular dystrophy pipeline development activities?
- What is the current limb girdle muscular dystrophy commercial assessment?
- What are the opportunities and challenges present in the limb girdle muscular dystrophy pipeline landscape?
- What is the efficacy and safety profile of limb girdle muscular dystrophy pipeline drugs?
- Which company is conducting major trials for limb girdle muscular dystrophy drugs?
- Which companies/institutions are involved in limb girdle muscular dystrophy collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in limb girdle muscular dystrophy?
Reasons To Buy This Report
The Limb Girdle Muscular Dystrophy Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for limb girdle muscular dystrophy. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into limb girdle muscular dystrophy collaborations, regulatory environments, and potential growth opportunities.
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Report Summary
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