지대형 근이영양증(LGMD) 시장 : 시장 인사이트, 역학, 시장 예측(2034)

Key Highlights:

• Limb-girdle muscular dystrophy (LGMD) is a diverse group of disorders with many subtypes categorized by disease gene and inheritance, it usually manifests in the proximal muscles around the hips and shoulders.
• According to DelveInsight's estimates, the total prevalent cases of Limb Girdle Muscular Dystrophy affects males and females in equal numbers. The prevalence of Limb Girdle Muscular Dystrophy is unknown, but estimates range from one in 14,500 to one in 123,000 and is expected to rise during the study period [2020-2034]. Estimates show the highest prevalent cases of Limb Girdle Muscular Dystrophy is in the United States followed by Europe.
• The United States accounts for the largest market size of Limb Girdle Muscular Dystrophy, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
• According to the findings, the age of onset can vary greatly even among individuals of the same family. The relative frequencies of the different types of LGMD vary from case to case, but worldwide LGMD2G, 2H and 2J are extremely rare.
• Currently, there no approved therapies but several pharma and biotechnology companies like Bridge Bio, Sarepta Therapeutics, and Asklepios Biopharmaceuticals, are actively working in the Limb Girdle Muscular Dystrophy (LGMD) market with their potential emerging pipeline drugs i.e. BBP-418, SRP-9003, and LION-101.

DelveInsight's "Limb Girdle Muscular Dystrophy (LGMD) - Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of Limb Girdle Muscular Dystrophy (LGMD), historical and forecasted epidemiology as well as the Limb Girdle Muscular Dystrophy (LGMD) market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

Limb Girdle Muscular Dystrophy (LGMD) market report provides real-world prescription pattern analysis, emerging drugs, market share of individual therapies, and historical and forecasted 7MM Limb Girdle Muscular Dystrophy (LGMD) market size from 2020 to 2034. The report also covers current Limb Girdle Muscular Dystrophy (LGMD) treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.

Geography Covered:

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Limb Girdle Muscular Dystrophy (LGMD) Understanding and Treatment Algorithm

Limb Girdle Muscular Dystrophy (LGMD) Overview, Country-Specific Treatment Guidelines and Diagnosis

Muscular dystrophy is a group of conditions causing muscle damage and weakness over time due to a lack of dystrophin, essential for normal muscle function. This deficiency can lead to difficulties with walking, swallowing, and muscle coordination.

Limb-girdle muscular dystrophy (LGMD), also known as pelvofemoral or proximal muscular dystrophy, is a rare, progressive genetic disorder. It is characterized by wasting and weakness of voluntary muscles in the hip and shoulder areas. The muscle weakness and atrophy are progressive and may spread to other muscles in the body.

There are two major groups of LGMDs. Called LGMD1 and LGMD2, these two groups are classified by the respective inheritance patterns: autosomal dominant and autosomal recessive. If one copy of the abnormal gene is sufficient to cause the disease, it is said to be autosomal dominant; if two copies are needed, then the inheritance pattern is autosomal recessive.

The Limb Girdle Muscular Dystrophy (LGMD) report provides an overview of Limb Girdle Muscular Dystrophy (LGMD) pathophysiology, diagnostic approaches, and detailed treatment algorithm along with a real-world scenario of a patient's journey beginning from the first symptom, the time taken for diagnosis to the entire treatment process.

Limb Girdle Muscular Dystrophy (LGMD) Treatment

Treatment for limb-girdle muscular dystrophy may include:

• Medications to relieve symptoms
• Respiratory therapy for breathing issues
• Occupational therapy for swallowing difficulties
• Diet and nutrition counseling for patients with difficulty chewing or swallowing

Limb Girdle Muscular Dystrophy (LGMD) Epidemiology

The Limb Girdle Muscular Dystrophy (LGMD) epidemiology chapter in the report provides historical as well as forecasted in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2024 to 2034. The Limb Girdle Muscular Dystrophy epidemiology is segmented with detailed insights into Total Prevalent Cases of Limb Girdle Muscular Dystrophy, Type-specific Prevalent Cases of Limb Girdle Muscular Dystrophy, Subtype-specific Prevalent Cases of Limb Girdle Muscular Dystrophy, Diagnosed and Treatable Cases of Limb Girdle Muscular Dystrophy [2020-2034]

• It is estimated LGMD is the fourth most common muscular dystrophy, with a pooled prevalence of 1.63 per 100,000 people, following myotonic dystrophy, dystrophinopathy, and facioscapulohumeral dystrophy. The prevalence varies among subtypes and regions.
• As per DelveInsight's estimates, LGMD prevalence across Europe ranges from 0.8 to 6 per 100,000.
• According to the findings, in the UK, dominantly inherited LGMD is less common and reported to be only 5-10% of all LGMD. It is also suggested that within the population of Northern England, LGMD accounts for 6.2% of all neuromuscular patients.

Limb Girdle Muscular Dystrophy (LGMD) Drug Chapters

The drug chapter segment of the Limb Girdle Muscular Dystrophy (LGMD) report encloses a detailed analysis of Limb Girdle Muscular Dystrophy (LGMD) late-stage (Phase III and Phase II) pipeline drugs. It also deep dives into the Limb Girdle Muscular Dystrophy (LGMD) pivotal clinical trial details, recent and expected market approvals, patent details, the latest news, and recent deals and collaborations.

Emerging Drugs

BBP-418: Bridge Bio

BridgeBio is pioneering an oral therapy, BBP-418, for limb girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). This treatment targets the dysfunctional alpha-dystroglycan protein, addressing the underlying cause of muscle weakness. The Phase II data shows potential for functional improvements and reduced muscle breakdown.

BBP-418 has received Fast Track and Orphan Drug designations from FDA and is currently in Phase III clinical trials.

SRP-9003: Sarepta Therapeutics

Sarepta Therapeutics is actively involved in the development of gene therapies for Limb-Girdle Muscular Dystrophy (LGMD). The company has multiple LGMD gene therapy programs in progress, including LGMD2E/R4, LGMD2D, LGMD2C, LGMD2B, LGMD2L, and LGMD2A, collectively addressing over 70% of known LGMD cases.

The drug is currently in Phase III developmental stage.

AB-1003 (LION-101): Asklepios Biopharmaceuticals/ Ask Bio

Asklepios Biopharmaceuticals' LION-101 is a cutting-edge therapy designed to combat LGMD by delivering a functional version of the FKRP gene to the body's cells. This innovative approach utilizes a recombinant adeno-associated virus, a viral vector known for its safety and ease of manipulation in laboratory settings.

AB-1003 has secured Investigational New Drug (IND) clearance and Fast Track designation from the US FDA. It is currently advancing through Phase I/II clinical trials.

Limb Girdle Muscular Dystrophy (LGMD) Market Outlook

Key players, such as Bridge Bio, Sarepta Therapeutics, and Asklepios Biopharmaceuticals are evaluating their lead candidates in different stages of clinical development, respectively. They aim to investigate their products for the treatment of Limb Girdle Muscular Dystrophy (LGMD).

The market size of Limb Girdle Muscular Dystrophy (LGMD) in the seven major markets is expected to increase substantially from 2024.

• There are currently no curative treatments for Limb Girdle Muscular Dystrophy; management primarily focuses on supportive care, physical therapy, and symptom relief.
• Increasing awareness of Limb Girdle Muscular Dystrophy among healthcare professionals and the public can lead to earlier diagnosis and intervention.
• The Limb Girdle Muscular Dystrophy market is still emerging, with a limited number of companies actively developing treatments. However, increasing interest in rare diseases and advances in biotechnology may attract more players.

Limb Girdle Muscular Dystrophy (LGMD) Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2024-2034, which depends on the competitive landscape, safety, and efficacy data along with order of entry. It is important to understand that the key players evaluating their novel therapies in the pivotal and confirmatory trials should remain vigilant when selecting appropriate comparators to stand the greatest chance of a positive opinion from regulatory bodies, leading to approval, smooth launch, and rapid uptake.

Limb Girdle Muscular Dystrophy (LGMD) Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II stages. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Limb Girdle Muscular Dystrophy (LGMD) emerging therapies.

KOL Views

To keep up with the real-world scenario in current and emerging market trends, we take opinions from Key Industry leaders working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts were contacted for insights on the evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility.

DelveInsight's analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as Nationwide Children's Hospital, Columbus, OH, the United States, University Hospitals Dorset NHS Foundation Trust, North Shore University Health System, Evanston, Illinois, etc., were contacted. Their opinion helps understand and validate current and emerging treatment patterns of Limb Girdle Muscular Dystrophy (LGMD). This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of gaps in disease diagnosis, patient awareness, physician acceptability, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint Analysis analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in event-free survival, one of the most important primary outcome measures is event-free survival and overall survival.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

Reimbursement of rare disease therapies can be limited due to lack of supporting policies and funding, challenges of high prices, lack of specific approaches to evaluating rare disease drugs given limited evidence, and payers' concerns about budget impact. The high cost of rare disease drugs usually has a limited effect on the budget due to the small number of eligible patients being prescribed the drug. The US FDA has approved several rare disease therapies in recent years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products independently.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report:

• The report covers a segment of key events, an executive summary, descriptive overview of Limb Girdle Muscular Dystrophy (LGMD), explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country specific treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging therapies, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the Limb Girdle Muscular Dystrophy (LGMD) market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Limb Girdle Muscular Dystrophy (LGMD) market.

Limb Girdle Muscular Dystrophy (LGMD) Report Insights

• Patient Cases
• Therapeutic Approaches
• Limb Girdle Muscular Dystrophy (LGMD) Pipeline Analysis
• Limb Girdle Muscular Dystrophy (LGMD) Market Size and Trends
• Existing and future Market Opportunity

Limb Girdle Muscular Dystrophy (LGMD) Report Key Strengths

• Ten Years Forecast
• 7MM Coverage
• Limb Girdle Muscular Dystrophy (LGMD) Epidemiology Segmentation
• Inclusion of Country specific treatment guidelines
• KOL's feedback on emerging therapies
• Key Cross Competition
• Conjoint analysis
• Drugs Uptake and Key Market Forecast Assumptions

Limb Girdle Muscular Dystrophy (LGMD) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

• What is the growth rate of the 7MM Limb Girdle Muscular Dystrophy (LGMD) treatment market?
• What was the Limb Girdle Muscular Dystrophy (LGMD) total market size, the market size by therapies, market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors/key catalysts for this growth?
• Is there any unexplored patient setting that can open the window for growth in the future?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends? Although multiple expert guidelines recommend testing for targetable mutations before therapy initiation, why do barriers to testing remain high?
• What are the current and emerging options for the treatment of Limb Girdle Muscular Dystrophy?
• How many companies are developing therapies for the treatment of Limb Girdle Muscular Dystrophy (LGMD)?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Limb Girdle Muscular Dystrophy (LGMD) Market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary of Limb Girdle Muscular Dystrophy (LGMD)

4. Key Events

5. Epidemiology and Market Forecast Methodology

6. Limb Girdle Muscular Dystrophy (LGMD) Market Overview at a Glance

• 6.1. Market Share (%) Distribution of Limb Girdle Muscular Dystrophy (LGMD) in 2020
• 6.2. Market Share (%) Distribution of Limb Girdle Muscular Dystrophy (LGMD) in 2034

7. Disease Background and Overview: Limb Girdle Muscular Dystrophy (LGMD)

• 7.1. Introduction
• 7.2. Clinical Characteristics of Limb Girdle Muscular Dystrophy (LGMD)
• 7.3. Symptoms
• 7.4. Complications and risk factors
• 7.5. Diagnosis
• 7.6. Establishing the Diagnosis
• 7.7. Differential diagnosis

8. Diagnostic Algorithm for Limb Girdle Muscular Dystrophy (LGMD)

9. Epidemiology and Patient Cases: 7MM

• 9.1. Key Findings
• 9.2. Assumptions and Rationale
• 9.3. Total Prevalent Cases of Limb Girdle Muscular Dystrophy (LGMD) in the 7MM
• 9.4. United States
  • 9.4.1. Total Prevalent Cases of Limb Girdle Muscular Dystrophy in the United States
  • 9.4.2. Type-specific Prevalent Cases of Limb Girdle Muscular Dystrophy in the United States
  • 9.4.3. Sub-type specific Prevalent Cases of Limb Girdle Muscular Dystrophy in the United States
  • 9.4.4. Diagnosed and Treatable Cases of Limb Girdle Muscular Dystrophy in the United States
• 9.5. EU4 and the UK
  • 9.5.1. Total Diagnosed Prevalent Cases of Limb Girdle Muscular Dystrophy (LGMD) in EU4 and the UK
  • 9.5.2. Type-specific Prevalent Cases of Limb Girdle Muscular Dystrophy in EU4 and the UK
  • 9.5.3. Sub-type specific Prevalent Cases of Limb Girdle Muscular Dystrophy in EU4 and the UK
  • 9.5.4. Diagnosed and Treatable Cases of Limb Girdle Muscular Dystrophy in EU4 and the UK
• 9.6. Japan
  • 9.6.1. Total Diagnosed Prevalent Cases of Limb Girdle Muscular Dystrophy (LGMD) in Japan
  • 9.6.2. Type-specific Prevalent Cases of Limb Girdle Muscular Dystrophy (LGMD) in Japan
  • 9.6.3. Sub-type specific Prevalent Cases of Limb Girdle Muscular Dystrophy in Japan
  • 9.6.4. Diagnosed and Treatable Cases of Limb Girdle Muscular Dystrophy in Japan

10. Treatment

• 10.1. Symptomatic treatment
• 10.2. Prevention of Primary Manifestations
• 10.3. Prevention of Secondary Complications

11. Patient Journey

12. Unmet Needs

List to be continued in full report

13. Emerging Drugs

• 13.1. Key Cross Competition
• 13.2. BBP-418: Bridge Bio
  • 13.2.1. Product Description
  • 13.2.2. Other Development Activities
  • 13.2.3. Clinical Development
  • 13.2.4. Safety and Efficacy
  • 13.2.5. Product Profile
• 13.3. SRP-9003: Sarepta Therapeutics
  • 13.3.1. Product Description
  • 13.3.2. Other Development Activities
  • 13.3.3. Clinical Development
  • 13.3.4. Safety and Efficacy
  • 13.3.5. Product Profile
• 13.4. AB-1003: Asklepios Biopharmaceuticals
  • 13.4.1. Product Description
  • 13.4.2. Other Development Activities
  • 13.4.3. Clinical Development
  • 13.4.4. Safety and Efficacy
  • 13.4.5. Product Profile

List to be continued in full report

14. Limb Girdle Muscular Dystrophy (LGMD): 7 Major Market Analysis

• 14.1. Key Findings
• 14.2. Market Outlook
• 14.3. Key Market Forecast Assumptions
• 14.4. Conjoint Analysis
• 14.6. Market Size of Limb Girdle Muscular Dystrophy (LGMD) in the 7MM
• 14.7. United States Market Size
  • 14.7.1. Total Market Size of Limb Girdle Muscular Dystrophy (LGMD) in the US
  • 14.7.2. Market Size by Therapies of Limb Girdle Muscular Dystrophy (LGMD) in the US
• 14.8. EU4 and the UK Market Size
  • 14.8.1. Total Market size of Limb Girdle Muscular Dystrophy (LGMD) in EU4 and the UK
  • 14.8.2. Market Size by Therapies of Limb Girdle Muscular Dystrophy (LGMD) in EU4 and the UK
• 14.9. Japan Market Size
  • 14.9.1. Total Market Size of Limb Girdle Muscular Dystrophy (LGMD) in Japan
  • 14.9.2. Market Size by Therapies in Japan

15. KOL Views

16. SWOT Analysis

17. Appendix

• 17.1. Bibliography
• 17.2. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight