시장보고서
상품코드
1147332

근긴장성이영양증 시장 : 시장 인사이트, 역학, 시장 예측(-2032년)

Myotonic Dystrophy - Market Insight, Epidemiology And Market Forecast - 2032

발행일: | 리서치사: DelveInsight Business Research LLP | 페이지 정보: 영문 177 Pages | 배송안내 : 1-2일 (영업일 기준)

※ 본 상품은 영문 자료로 한글과 영문 목차에 불일치하는 내용이 있을 경우 영문을 우선합니다. 정확한 검토를 위해 영문목차를 참고해주시기 바랍니다.

주요 7개국의 근긴장성이영양증 시장 규모는 2021년에 8,000만 달러로 평가되었습니다. 이 시장은 앞으로도 한층 더 확대될 것으로 예측됩니다.

주요 7개국의 근긴장성이영양증(Myotonic Dystrophy) 시장에 대해 조사했으며, 시장 개요와 함께 역학, 환자 동향, 새로운 치료법, 2032년까지 시장 규모 예측 및 의료 미충족 요구 등의 정보를 제공합니다.

목차

제1장 주요 인사이트

제2장 보고서 서론

제3장 근긴장성이영양증 시장 개요

제4장 근긴장성이영양증 개요

제5장 질환의 배경과 개요

  • 서론
  • 임상 증상
  • 분류
  • 병인
  • 병태생리학
  • 진단
  • 치료

제6장 역학과 환자수

  • 주요 조사 결과
  • 주요 7개국의 진단 환자수 합계
  • 가정과 근거
  • 미국
  • 유럽 5개국
  • 일본

제7장 환자 동향

제8장 새로운 치료법

제9장 기타 자산

제10장 근긴장성이영양증(DM) : 주요 7개국 분석

  • 주요 조사 결과
  • 주요 7개국의 DM 전체 시장 규모
  • 시장 전망
  • 속성 분석
  • 미국 시장 규모
  • 유럽 5개국 시장 규모
  • 일본 시장 규모

제11장 SWOT 분석

제12장 KOL(Key Opinion Leader)의 견해

제13장 미충족 요구

제14장 시장 접근

제15장 두자어와 약어

제16장 부록

제17장 DelveInsight의 서비스 내용

제18장 면책사항

제19장 DelveInsight에 대해

LSH 22.11.11

DelveInsight's 'Myotonic Dystrophy (DM) - Market Insights, Epidemiology, and Market Forecast - 2032' report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of DM in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

The myotonic dystrophy (DM) market report provides current treatment practices, emerging drugs, market share of the individual therapies, and current and forecasted myotonic dystrophy (DM) market size from 2019 to 2032, segmented by 7MM. The report also covers the current myotonic dystrophy (DM) treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study period: 2019-2032.

Myotonic Dystrophy (DM): Disease Understanding and Treatment Algorithm

Myotonic Dystrophy (DM) overview

According to the National Organization for Rare Disorders (NORD), myotonic dystrophy (DM) refers to a group of two rare genetic (autosomal dominant) disorders of muscle affecting multiple systems of the body; characterized by a clinical triad of progressive weakness, myotonia, and early-onset cataracts. DM is a type of muscular dystrophy. Muscular dystrophies are characterized by weakness and degeneration of various voluntary muscles of the body. Specific abnormalities characterize each disorder (e.g., variation of muscle fiber size, muscle fiber necrosis, scar tissue formation, and inflammation) in muscle biopsy from patients. There are two main types of DM- DM type 1 (DM1) and DM type 2 (DM2). DM1 is also known as Steinert disease, named after Dr. Steinert, who, along with colleagues, first described the classic form in the medical literature in 1909. DM2 is also known as Ricker syndrome or proximal DM or PROMM.

A change or alteration causes DM1 in the myotonic dystrophy protein kinase (DMPK) gene. DM2 is caused by a change or alteration in the nucleic acid-binding protein (CNBP) gene, also called the ZNF9 gene. It is caused when a certain segment of DNA at the end of a gene is abnormally repeated many times, forming an unstable region in the gene, and is called a triplet repeat expansion. Unaffected individuals have a small number of repeats (up to about 35) in this region, but in affected individuals, the number can be much higher- rising to several thousand in congenitally affected population; DM1 is estimated to affect about 1 in 8,000-20,000 people in the general population. However, the prevalence of DM1 and DM2 vary greatly among different countries and ethnic groups.

Diagnosis of DM may be suspected based upon a thorough clinical evaluation, a detailed patient and family history, and identifying characteristic physical findings. A family history of muscle weakness and myotonia strongly indicates a DM diagnosis. Several laboratory tests can clarify the clinical diagnosis of DM, including blood tests, electromyography (EMG), magnetic resonance imaging (MRI), muscle biopsy, and genetic testing. Though different diagnostic options are available, the definitive test for DM is a genetic test.

Currently, no approved treatment offers a permanent cure for DM, but researchers are studying ways to help people with these disorders. Current treatment is directed toward the specific symptoms that are apparent in each individual. Pharmacological management of DM involves the usage of pain medications like nonsteroidal anti-inflammatories (NSAIDs), gabapentin, tricyclic antidepressants, mexiletine, and low-doses of glucocorticoids such as prednisone. Surgical procedures are opted for removing cataracts if they affect vision. However, there have been reports that cataracts can recur after removal. Some people with breathing problems during sleep may require noninvasive ventilation, which involves breathing support with a mask or similar device. Medical devices like a pacemaker or implantable cardioverter defibrillator (ICD) are advised for individuals with cardiological complications.

Myotonic Dystrophy (DM) Diagnosis and Treatment

It covers the details of conventional and current medical therapies and diagnoses available in the myotonic dystrophy (DM) market to treat the condition. It also provides country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

Myotonic Dystrophy (DM) Epidemiology

The myotonic dystrophy (DM) epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every 7MM. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of KOL. The report also provides the prevalent patient pool, trends, and assumptions.

Key Findings

The disease epidemiology covered in the report provides historical and forecasted myotonic dystrophy (DM) epidemiology segmented as the diagnosed prevalent cases of myotonic dystrophy (DM), type-specific diagnosed cases of myotonic dystrophy (DM), type-specific diagnosed cases of DM1, age-specific diagnosed cases of DM, and comorbidity associated diagnosed cases with myotonic dystrophy (DM). The report includes the prevalent myotonic dystrophy (DM) scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise Myotonic Dystrophy (DM) Epidemiology

The epidemiology segment also provides the myotonic dystrophy (DM) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The total diagnosed prevalent patient population of myotonic dystrophy (DM) in the 7MM countries was close to 80,000 cases in 2021.

As per the estimates, the US had the highest patient population of myotonic dystrophy (DM) in 2021. Among the EU5 countries, Germany had the highest diagnosed patient population of myotonic dystrophy (DM), with over 9,000 cases, followed by France in 2021. On the other hand, Spain had the lowest diagnosed prevalent patient population of myotonic dystrophy (DM), with approximately 6,000 cases in 2021.

Myotonic Dystrophy (DM) Drug Chapters

The drug chapter segment of the myotonic dystrophy (DM) report encloses the detailed analysis of myotonic dystrophy (DM) marketed drugs and late-stage (Phase III, Phase II/III, Phase II, and Phase I/II) pipeline drugs. It also helps understand the myotonic dystrophy (DM) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

The current standard of care lacks disease-modifying therapy. Management consists primarily of monitoring for complications and standard of care (assistive devices, hormone therapy, and pain medication). A few clinical studies have systematically evaluated the use of therapeutic agents in DM. The lack of scientific evidence paired with the multi-systematic and highly variable presentation of the disease makes identifying and selecting appropriate medications particularly challenging for prescribing physicians.

Thus, there is an urgent need to identify existing medications that are potentially safe and effective for DM patients and evaluate these therapies using appropriate clinical trials.

Products detail in the report…

Myotonic Dystrophy (DM) Emerging Drugs

Drug developers are gradually shifting their attention toward myotonic dystrophy (DM) to meet the patient pool's current demands and counter the unmet needs of the therapeutic market.

Several companies are working robustly on many new therapies, such as AMO-02 (AMO Pharma Limited), mexiletine (Lupin Ltd.), and pitolisant (Harmony Biosciences, LLC).

AMO-02 (AMO Pharma Limited) is in development for treating congenital myotonic dystrophy (CDM) and has the potential for use in additional CNS, neuromuscular and other orphan indications. It has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3B. Currently, the company conducts two Phase II/III studies in children and adolescents with CDM.

Products detail in the report…

Myotonic Dystrophy (DM) Market Outlook

The myotonic dystrophy (DM) market outlook of the report builds a detailed comprehension of the historical, current, and forecasted myotonic dystrophy (DM) market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.

This segment gives a thorough detail of the myotonic dystrophy (DM) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market, and KOL view. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

As per DelveInsight, the myotonic dystrophy (DM) market in 7MM is expected to change in 2019-2032.

Key Findings

This section includes a glimpse of the myotonic dystrophy (DM) market in 7MM. In 2021, the myotonic dystrophy (DM) market size was approximately USD 80 million in the 7MM.

The United States: Market Outlook

This section provides the total myotonic dystrophy (DM) market size. It also provides the market size of myotonic dystrophy (DM) by therapies in the United States.

The United States accounts for the highest market size of myotonic dystrophy (DM) than the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

EU5 Countries: Market Outlook

This section provides the total myotonic dystrophy (DM) market size. It also provides myotonic Dystrophy (DM) market size by therapies in Germany, France, Italy, Spain, and the United Kingdom.

Japan Market Outlook

This section provides the total myotonic dystrophy (DM) market size. It also provides the market size of myotonic dystrophy (DM) by therapies in Japan.

Myotonic Dystrophy (DM) Drugs Uptake

This section focuses on the uptake rate of potential drugs launched or expected to be launched in the market during 2019-2032. The analysis covers myotonic dystrophy (DM) market uptake by drugs, patient uptake by therapies, and sales of each drug.

This helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and making financial and regulatory decisions.

Myotonic Dystrophy (DM) Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II. It also analyses myotonic dystrophy (DM)'s key players in developing targeted therapeutics.

Major players include: AMO Pharma Limited, Lupin Ltd., Harmony Biosciences, and others whose key products are expected to get launched in the US market by 20XX.

Pipeline Development Activities

The report covers collaborations, acquisitions, mergers, licensing, and patent details for emerging myotonic dystrophy (DM) therapies.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the myotonic dystrophy (DM) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging treatment patterns or myotonic dystrophy (DM) market trends. This will support the clients in potential novel treatments by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform a competitive and market intelligence analysis of the myotonic dystrophy (DM) market using various competitive intelligence tools: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report:

  • The report covers the descriptive overview of myotonic dystrophy (DM), explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
  • Comprehensive insight is provided into the myotonic dystrophy (DM) epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for myotonic dystrophy (DM) is provided, along with the assessment of new therapies that will impact the current treatment landscape
  • A detailed review of the myotonic dystrophy (DM) market; historical and forecasted, is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies by understanding trends shaping and driving the global myotonic dystrophy (DM) market

Report Highlights:

  • Recently, the myotonic dystrophy (DM) market is set to change due to the rising awareness of the disease and incremental healthcare spending worldwide; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence myotonic dystrophy (DM) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
  • Major players are involved in developing therapies for myotonic dystrophy (DM). The launch of emerging therapies will significantly impact the myotonic Dystrophy (DM) market
  • For myotonic dystrophy (DM), a better understanding of disease pathogenesis will also contribute to developing novel therapeutics
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the R&D activities

Myotonic Dystrophy (DM) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Myotonic Dystrophy (DM) Pipeline Analysis
  • Myotonic Dystrophy (DM) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Myotonic Dystrophy (DM) Report Key Strengths

  • 11-year Forecast
  • 7MM Coverage
  • Myotonic Dystrophy (DM) Epidemiology Segmentation
  • Key Competitors
  • Highly Analyzed Market
  • Drugs Uptake

Myotonic Dystrophy (DM) Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness

Key Questions

Market Insights:

  • What was the market share percentage distribution, and how would it look by 2032?
  • What would be the total market size and market size of myotonic dystrophy (DM) by therapies across the 7MM forecast period (2019-2032)?
  • What are the key findings about the market across 7MM, and which country will have the largest myotonic dystrophy (DM) market size during the forecast period (2019-2032)?
  • At what CAGR is the myotonic dystrophy (DM) market expected to grow in the 7MM forecast period (2019-2032)?
  • What would be the myotonic dystrophy (DM) market outlook across the 7MM forecast period (2019-2032)?
  • What would be the myotonic dystrophy (DM) market growth until 2032 and the resultant market size by 2032?
  • How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

  • What are the disease risk, burdens, and unmet needs of myotonic dystrophy (DM)?
  • What is the historical patient pool of myotonic dystrophy (DM) covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of myotonic dystrophy (DM) covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What will be the growth opportunities in the 7MM concerning the patient population of myotonic dystrophy (DM)?
  • Out of all the 7MM, which country would have the highest prevalence of myotonic dystrophy (DM) during the forecast period (2019-2032)?
  • At what CAGR is the population expected to grow in the 7MM forecast period (2019-2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

  • What are the current options to treat myotonic dystrophy (DM)?
  • What are the current treatment guidelines for treating myotonic dystrophy (DM) in the US, Europe, and Japan?
  • What are the myotonic dystrophy (DM) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, efficacy, etc.?
  • How many companies are developing therapies to treat myotonic dystrophy (DM)?
  • How many therapies are developed by each company to treat myotonic dystrophy (DM)?
  • How many emerging therapies are in the mid-stage and late stages of development to treat myotonic dystrophy (DM)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and licensing activities related to myotonic dystrophy (DM) therapies?
  • What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies for myotonic dystrophy (DM) and its status?
  • Which key designations have been granted for the emerging therapies for myotonic dystrophy (DM)?
  • What are the global historical and forecasted myotonic dystrophy (DM) markets?

Reasons to buy:

  • The report will help in developing business strategies by understanding trends shaping and driving the myotonic dystrophy (DM) market
  • To understand the future market competition in the myotonic dystrophy (DM) market and an insightful review of the key market drivers and barriers
  • Organize sales and marketing efforts by identifying the best opportunities for myotonic dystrophy (DM) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan
  • Identifying upcoming solid players in the market will help devise strategies that will help get ahead of competitors
  • Organize sales and marketing efforts by identifying the best opportunities for the myotonic dystrophy (DM) market
  • To understand the future market competition in the myotonic dystrophy (DM) market

Table of Contents

1. Key Insights

2. Report Introduction

3. Myotonic Dystrophy Market Overview at a Glance

  • 3.1. Market Share (%) Distribution of Myotonic Dystrophy in 2019
  • 3.2. Market Share (%) Distribution Myotonic Dystrophy in 2032

4. Executive Summary of Myotonic Dystrophy

5. Disease background and overview

  • 5.1. Introduction
  • 5.2. Clinical Manifestations
  • 5.3. Classification
  • 5.4. Etiology
  • 5.5. Pathophysiology
  • 5.6. Diagnosis
    • 5.6.1. Differential diagnosis
  • 5.7. Treatment
    • 5.7.1. Treatment guidelines
      • 5.7.1.1. Management of respiratory complications in DM
      • 5.7.1.2. Management of cardiovascular complications in DM
      • 5.7.1.3. Pregnancy and obstetrics management
      • 5.7.1.4. Management of skeletal muscle weakness and rehabilitation
      • 5.7.1.5. Management of ocular complications
      • 5.7.1.6. Management of gastrointestinal complications
      • 5.7.1.7. Management of gastrointestinal complications
      • 5.7.1.8. Management of neuropsychiatric complications and excessive daytime sleepiness
      • 5.7.1.9. Management of endocrine and metabolic complications
      • 5.7.1.10. Recommendations for surgery and anesthesia in DM patients
      • 5.7.1.11. Management of CDM1
      • 5.7.1.12. Genetic counseling

6. Epidemiology and Patient Population

  • 6.1. Key Findings
  • 6.2. Total Diagnosed Prevalent Cases of DM in the 7MM
  • 6.3. Assumption and Rationale
  • 6.4. The United States
    • 6.4.1. Diagnosed Prevalence of DM in the United States
    • 6.4.2. Type-Specific cases of DM in the United States
    • 6.4.3. Type-Specific Cases of DM1 in the United States
    • 6.4.4. Age-Specific Diagnosed Cases of DM in the United States
    • 6.4.5. Comorbidity associated Diagnosed Cases with DM in the United States
  • 6.5. The EU5
    • 6.5.1. Diagnosed prevalence of DM in the EU5
    • 6.5.2. Type-specific Diagnosed Cases of DM in the EU5
    • 6.5.3. Type-specific Diagnosed Cases of DM1 in the EU5
    • 6.5.4. Age-specific Diagnosed Cases of DM in the EU5
    • 6.5.5. Comorbidity associated Diagnosed Cases with DM in the EU5
  • 6.6. Japan
    • 6.6.1. Diagnosed Prevalence of DM in Japan
    • 6.6.2. Type-specific Diagnosed Cases of DM in Japan
    • 6.6.3. Type-specific Diagnosed Cases of DM1 in Japan
    • 6.6.4. Age-specific Diagnosed Cases of DM in Japan
    • 6.6.5. Comorbidity associated Diagnosed Cases with DM in Japan

7. Patient Journey

8. Emerging Therapies

  • 8.1. Key cross: Emerging
  • 8.2. AMO-02 (tideglusib): AMO Pharma Limited
    • 8.2.1. Drug description
    • 8.2.2. Other development activities
    • 8.2.3. Clinical development
      • 8.2.3.1. Clinical trial information
    • 8.2.4. Safety and efficacy
    • 8.2.5. Product profile
    • 8.2.6. Analyst comments
  • 8.3. Mexiletine: Lupin Ltd.
    • 8.3.1. Drug description
    • 8.3.2. Other development activities
    • 8.3.3. Clinical development
      • 8.3.3.1. Clinical trial information
    • 8.3.4. Product profile
    • 8.3.5. Analyst comment
  • 8.4. Pitolisant: Harmony Biosciences, LLC
    • 8.4.1. Drug description
    • 8.4.2. Other development activities
    • 8.4.3. Clinical development
      • 8.4.3.1. Clinical trial information
    • 8.4.4. Product profile
    • 8.4.5. Analyst comment

9. Other Assets

  • 9.1. Key cross: Other Assets
  • 9.2. AOC 1001: Avidity Biosciences, Inc.
    • 9.2.1. Drug description
    • 9.2.2. Other development activities
    • 9.2.3. Clinical development
      • 9.2.3.1. Clinical trial information
    • 9.2.4. Product profile
  • 9.3. DYNE-101: Dyne Therapeutics
    • 9.3.1. Drug description
    • 9.3.2. Other development activities
    • 9.3.3. Clinical development
      • 9.3.3.1. Clinical trial information
    • 9.3.4. Product profile

10. Myotonic Dystrophy (DM): 7MM Analysis

  • 10.1. Key Findings
  • 10.2. Total Market Size of DM in the 7MM
  • 10.3. Market Outlook
  • 10.4. Attribute Analysis
  • 10.5. The United States Market Size
    • 10.5.1. Total Market Size of DM in the United States
    • 10.5.2. Market Size of DM by Therapies in the United States
  • 10.6. The EU5 Market Size
    • 10.6.1. Total Market Size of DM in the EU5
    • 10.6.2. Market Size of DM in the EU5 by Therapies
  • 10.7. Japan Market Size
    • 10.7.1. Total Market Size of DM in Japan
    • 10.7.2. Market Size of DM in Japan by Therapies

11. SWOT Analysis

12. KOL Views

13. Unmet Need

14. Market Access

15. Acronyms and Abbreviations

16. Appendix

  • 16.1. Bibliography
  • 16.2. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

19. About DelveInsight

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