市場調査レポート

商品コード

534853

脊髄性筋萎縮症（SMA）- 市場考察、疫学、市場予測

Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast - 2028

出版日: 受注後作成 | 発行:

DelveInsight Business Research LLP | ページ情報: 英文 170 Pages | 納期: 2～10営業日

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全表示
概要
図表
目次

概要

サンプル依頼リストに入れる

主要7カ国における脊髄性筋萎縮症（SMA）の有病数は、2017年に3万8,900人と推定され、同年の脊髄性筋萎縮症（SMA）市場は8億2,500万米ドルと評価されています。

当レポートでは、脊髄性筋萎縮症（SMA）市場について調査し、疾病の概要、主要7カ国（米国、英国、ドイツ、フランス、イタリア、スペイン、日本）の市場動向、有病数および市場規模の予測（今後10年間分）、潜在成長性、成長要因および障壁の分析、上市済み/開発中の治療薬のプロファイル、現在のアンメットニーズなどを包括的にまとめています。

患者人口と予測パラメーター
重要点
主要7カ国の脊髄性筋萎縮症（SMA）有病数
主要7カ国の脊髄性筋萎縮症（SMA）罹患数（実績値）
主要7カ国の脊髄性筋萎縮症（SMA）罹患数（予測値）
米国
- 脊髄性筋萎縮症（SMA）罹患数
EU5カ国
- ドイツ
  - 脊髄性筋萎縮症（SMA）罹患数
- フランス
  - 脊髄性筋萎縮症（SMA）罹患数
- 英国
  - 脊髄性筋萎縮症（SMA）罹患数
- スペイン
  - 脊髄性筋萎縮症（SMA）罹患数
- イタリア
  - 脊髄性筋萎縮症（SMA）罹患数
日本
- 脊髄性筋萎縮症（SMA）罹患数
第5章治療のガイドライン
- 治療方針
- 適応外薬に対する専門家の意見
- 米国の団体
- 欧州の団体
- 日本の団体
第6章アンメットニーズ

第7章上市済みの治療薬
- 上市済みの治療薬
- Spinraza（nusinersen）：Biogen, Inc.
  - 薬物の説明
  - 作用機序
  - 規制のマイルストーン
  - 長所と短所
  - 安全性と有効性
  - 製品のプロファイル
  - 売上げ
第8章多元的競争

第9章開発中の新薬
- AVXS-101：Avexis
  - 規制のマイルストーン
  - 臨床開発
  - 進行中の臨床試験情報
  - 安全性と有効性
  - 長所と短所
  - 製品のプロファイル
- LMI070：Novartis Pharmaceuticals
- CK-2127107: Cytokinetics, Inc.
- RG7916: Hoffmann-La Roche
- Olesoxime (TRO19622): Hoffmann-La Roche

第10章その他の新治療法

第11章市場規模

主な所見
世界の脊髄性筋萎縮症（SMA）市場の分析
世界の脊髄性筋萎縮症（SMA）市場の見通し
既存の治療薬・上市予定の治療薬
主要7カ国の脊髄性筋萎縮症（SMA）市場
主要7カ国の脊髄性筋萎縮症（SMA）市場：治療薬別
主要7カ国で使用される治療薬のシェア

第12章米国

既存治療薬の動向
新しい治療薬と市場への影響
米国における脊髄性筋萎縮症（SMA）市場の予測
米国における脊髄性筋萎縮症（SMA）市場：治療薬別
米国における脊髄性筋萎縮症（SMA）患者数：治療薬別

第13章欧州

既存治療薬の動向
新しい治療薬と市場への影響
EU5カ国における脊髄性筋萎縮症（SMA）市場の予測
EU5カ国における脊髄性筋萎縮症（SMA）市場：治療薬別
EU5カ国における脊髄性筋萎縮症（SMA）患者数：治療薬別
ドイツ
フランス
英国
スペイン
イタリア
日本

第14章市場の成長要因と障壁

第15章付録

第16章調査手法

第17章コンサルティングサービス

第18章免責事項

第19章 DelveInsightについて

図表

List of Tables

Table 1 Types of SMA linked to Chromosome-5
Table 2 Total Population of Spinal Muscular Atrophy in the 7MM (2017-2028)
Table 3 Total Diagnosed Population of Spinal Muscular Atrophy in the 7MM (2017-2028)
Table 4 Spinal Muscular Atrophy Prevalent Cases in the United States (2017-2028)
Table 5 Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United States (2017-2028)
Table 6 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United States (2017-2028)
Table 7 Spinal Muscular Atrophy Mutation specific Prevalence in the United States (2017-2028)
Table 8 Spinal Muscular Atrophy Prevalent Cases in Germany (2017-2028)
Table 9 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Germany (2017-2028)
Table 10 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Germany (2017-2028)
Table 11 Spinal Muscular Atrophy Mutation specific Prevalence in Germany (2017-2028)
Table 12 Spinal Muscular Atrophy Prevalent Cases in France (2017-2028)
Table 13 Spinal Muscular Atrophy Diagnosed Prevalent Cases in France (2017-2028)
Table 14 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in France (2017-2028)
Table 15 Spinal Muscular Atrophy Mutation specific Prevalence in France (2017-2028)
Table 16 Spinal Muscular Atrophy Prevalent Cases in Italy (2017-2028)
Table 17 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Italy (2017-2028)
Table 18 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Italy (2017-2028)
Table 19 Spinal Muscular Atrophy Mutation specific Prevalence in Italy (2017-2028)
Table 20 Spinal Muscular Atrophy Prevalent Cases in Spain (2017-2028)
Table 21 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Spain (2017-2028)
Table 22 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Spain (2017-2028)
Table 23 Spinal Muscular Atrophy Mutation specific Prevalence in Spain (2017-2028)
Table 24 Spinal Muscular Atrophy Prevalent Cases in the United Kingdom (2017-2028)
Table 25 Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United Kingdom (2017-2028)
Table 26 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United Kingdom (2017-2028)
Table 27 Spinal Muscular Atrophy Mutation specific Prevalence in the United Kingdom (2017-2028)
Table 28 Spinal Muscular Atrophy Prevalent Cases in Japan (2017-2028)
Table 29 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Japan (2017-2028)
Table 30 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Japan (2017-2028)
Table 31 Spinal Muscular Atrophy Mutation specific Prevalence in Japan (2017-2028)
Table 32 Standards of care for pulmonary, gastrointestinal, nutritional, and orthopedic care and rehabilitation
Table 33 United States Association Current Recommendations for SMA
Table 34 European Association Current Recommendations for SMA
Table 35 Japanese Association Current Recommendations for SMA
Table 36 Comparison of emerging drugs under development
Table 37 Clinical Trial Description
Table 38 Clinical Trial Description
Table 39 Clinical Trial Description
Table 40 Clinical Trial Description
Table 41 Clinical Trial Description
Table 42 7 Major Market Size of Spinal Muscular Atrophy in USD Million (2017-2028)
Table 43 United States Market Size of Spinal Muscular Atrophy, USD Million (2017-2028)
Table 44 Market size of Spinal Muscular Atrophy by therapies in the United States, in USD Million (2017-2028)
Table 45 Market Size of Spinal Muscular Atrophy in Germany, in USD Million (2017-2028)
Table 46 Market size of Spinal Muscular Atrophy by therapies in Germany, in USD Million (2017-2028)
Table 47 Market Size of Spinal Muscular Atrophy in France, in USD Million (2017-2028)
Table 48 Market size of Spinal Muscular Atrophy by therapies in France, in USD Million (2017-2028)
Table 49 Market Size of Spinal Muscular Atrophy in Italy, in USD Million (2017-2028)
Table 50 Market size of Spinal Muscular Atrophy by therapies in Italy, in USD Million (2017-2028)
Table 51 Market Size of Spinal Muscular Atrophy in Spain, in USD Million (2017-2028)
Table 52 Market size of Spinal Muscular Atrophy by therapies in Spain, in USD Million (2017-2028)
Table 53 Market Size of Spinal Muscular Atrophy in the UK, in USD Million (2017-2028)
Table 54 Market size of Spinal Muscular Atrophy by therapies in the United Kingdom, in USD Million (2017-2028)
Table 55 Market Size of Spinal Muscular Atrophy in Japan, in USD Million (2017-2028)
Table 56 Market size of Spinal Muscular Atrophy by therapies in Japan, in USD Million (2017-2028)

List of Figures

Figure 1 Inheritance of SMA in an autosomal recessive pattern
Figure 2 Classification of SMA
Figure 3 Schematic representation of a portion of chromosome 5 that contains two SMN genes
Figure 4 Targets for therapy in SMA
Figure 5 Diagnostic Evaluation of SMA
Figure 6 Spinal Muscular Atrophy Prevalent Cases in the United States (2017-2028)
Figure 7 Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United States (2017-2028)
Figure 8 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United States (2017-2028)
Figure 9 Spinal Muscular Atrophy Mutation specific Prevalence in the United States (2017-2028)
Figure 10 Spinal Muscular Atrophy Prevalent Cases in Germany (2017-2028)
Figure 11 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Germany (2017-2028)
Figure 12 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Germany (2017-2028)
Figure 13 Spinal Muscular Atrophy Mutation specific Prevalence in Germany (2017-2028)
Figure 14 Spinal Muscular Atrophy Prevalent Cases in France (2017-2028)
Figure 15 Spinal Muscular Atrophy Diagnosed Prevalent Cases in France (2017-2028)
Figure 16 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in France (2017-2028)
Figure 17 Spinal Muscular Atrophy Mutation specific Prevalence in France (2017-2028)
Figure 18 Spinal Muscular Atrophy Prevalent Cases in Italy (2017-2028)
Figure 19 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Italy (2017-2028)
Figure 20 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Italy (2017-2028)
Figure 21 Spinal Muscular Atrophy Mutation specific Prevalence in Italy (2017-2028)
Figure 22 Spinal Muscular Atrophy Prevalent Cases in Spain(2017-2028)
Figure 23 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Spain (2017-2028)
Figure 24 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Spain (2017-2028)
Figure 25 Spinal Muscular Atrophy Mutation specific Prevalence in Spain (2017-2028)
Figure 26 Spinal Muscular Atrophy Prevalent Cases in the United Kingdom (2017-2028)
Figure 27 Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United Kingdom (2017-2028)
Figure 28 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United Kingdom (2017-2028)
Figure 29 Spinal Muscular Atrophy Mutation specific Prevalence in the United Kingdom (2017-2028)
Figure 30 Spinal Muscular Atrophy Prevalent Cases in Japan (2017-2028)
Figure 31 Spinal Muscular Atrophy Diagnosed Prevalent Cases in Japan (2017-2028)
Figure 32 Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Japan (2017-2028)
Figure 33 Spinal Muscular Atrophy Mutation specific Prevalence in Japan (2017-2028)
Figure 36 Sign & Symptoms of Spinal Muscular Atrophy
Figure 37 Anticipated Therapeutic Strategies
Figure 38 SMA Newborn Screening Treatment Schematic for SMN-Up-Regulating Therapy
Figure 39 Unmet Needs of Spinal Muscular Atrophy
Figure 40 7 Major Market Size of Spinal Muscular Atrophy in USD Million (2017-2028)
Figure 41 Market Size of Spinal Muscular Atrophy in the United States, USD Millions (2017-2028)
Figure 42 Market size of Spinal Muscular Atrophy by therapies in the US, in USD Million (2017-2028)
Figure 43 Market Size of Spinal Muscular Atrophy in Germany, USD Million (2017-2028)
Figure 44 Market Size of Spinal Muscular Atrophy by therapies, in Germany, in USD Million (2017-2028)
Figure 45 Market Size of Spinal Muscular Atrophy in France, USD Million (2017-2028)
Figure 46 Market Size of Spinal Muscular Atrophy by therapies, in France, in USD Million (2017-2028)
Figure 47 Market Size of Spinal Muscular Atrophy in Italy, USD Million (2017-2028)
Figure 48 Market Size of Spinal Muscular Atrophy by therapies in Italy, in USD Million (2017-2028)
Figure 49 Market Size of Spinal Muscular Atrophy in Spain, USD Million (2017-2028)
Figure 50 Market Size of Spinal Muscular Atrophy by therapies in Spain, in USD Million (2017-2028)
Figure 51 Market Size of Spinal Muscular Atrophy in the UK, USD Million (2017-2028)
Figure 52 Market Size of Spinal Muscular Atrophy by therapies in the UK, in USD Million (2017-2028)
Figure 53 Market Size of Spinal Muscular Atrophy in Japan, USD Million (2017-2028)
Figure 54 Market Size of Spinal Muscular Atrophy by therapies in Japan, in USD Million (2017-2028)
Figure 55 Market Drivers
Figure 56 Market Barriers

Product Code: DIMI0469

DelveInsight's 'Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast-2028' report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Spinal Muscular Atrophy in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.

The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Spinal Muscular Atrophy from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.

Geography Covered

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

Study Period: 2017-2028

Spinal Muscular Atrophy- Disease Understanding and Treatment Algorithm

According to the National Institute of Health (NIH), Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement. The weakness tends to be more severe in the muscles that are close to the center of the body (proximal) compared to muscles away from the body's center (distal). The muscle weakness usually worsens with age. SMA is one of the most common causes of infant death and mobility impairment. It mostly affects proximal muscles and respiratory muscles. There are three common forms of spinal muscular atrophy classified by age at onset and severity of functional impairment. Type 1 spinal muscular atrophy has been termed Werdnig-Hoffmann disease and is most usually fatal by 2 years of age. Children with spinal muscular atrophy type 2 can usually achieve the ability to sit, but have limited walking. Patients with type 3 or Kugelberg-Welander syndrome develop weakness in teen or young adult years.

The DelveInsight Spinal Muscular Atrophy market report gives the thorough understanding of the Spinal Muscular Atrophy by including details such as disease introduction, classification, causes, pathophysiology, biomarkers in Spinal Muscular Atrophy, symptoms, diagnosis and differential diagnosis. It also provides treatment algorithms and treatment guidelines for Spinal Muscular Atrophy in the US, Europe, and Japan.

Spinal Muscular Atrophy Epidemiology

The Spinal Muscular Atrophy epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalent Cases of Spinal Muscular Atrophy, Total Diagnosed Prevalent Cases of Spinal Muscular Atrophy, Type specific Diagnosed Prevalence of Spinal Muscular Atrophy, Mutation specific Prevalence of Spinal Muscular Atrophy) scenario of Spinal Muscular Atrophy in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2028.

According to a study conducted by R. Vamshi et al titled as "Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease" published in Journal of Managed Care and Specialty Pharmacy (JMCP) which stated the SMN1 gene product-SMN protein-is crucial for motor neuron development. In approximately 95% of patients, SMA results from homozygous deletion or conversion of SMN1. In about 2% of patients, de novo deletions occur in one of the SMN1 alleles; in 3%-4%, other mutations can be found, typically with an SMN1 deletion on the other allele.

Spinal Muscular Atrophy Drug Chapters

This segment of the Spinal Muscular Atrophy report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the 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.

Current management of SMA is based on supportive and multidisciplinary care with a focus on reducing complications and improving quality of life. Pulmonary disease is the major cause of mortality in SMA type I and II and affects a small proportion of patients with SMA type III. Management strategies include airway clearance, cough assistance, nocturnal non-invasive ventilatory support, and continuous non-invasive ventilation. The supportive approaches involve nutrition, orthopedic care, rehabilitation, long-term feeding tubes etc.

Spinraza, manufactured by Biogen is the only approved drug for the treatment of Spinal Muscular Atrophy in adults and children. The active ingredient of the drug is Nusinersen. The drug increases full-length survival motor neuron (SMN) protein by targeting the process through which it is produced by the SMN2 gene.

There is no cure, but treatments can improve some symptoms and in some cases, help the patients live longer. Researchers are working robustly to find new ways to fight the disease.

To meet the increasing demand for the treatment of Spinal Muscular Atrophy, companies have shifted their focus towards the development of targeted therapies. Expected launch of potential therapies may increase the market size in the coming years, assisted by an increase in the Incident population of Spinal Muscular Atrophy& awareness of the disease. The overall dynamics of Spinal Muscular Atrophy market is anticipated to change in the coming years owing to the expected launch of emerging therapies of the major key players such as Novartis, Hoffmann-La Roche, Astellas Pharma, Cytokinetics, and some others, will significantly increase the market during the forecast period (2019-2028).

Spinal Muscular Atrophy Market Outlook

The Spinal Muscular Atrophy market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

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

According to DelveInsight, the market of Spinal Muscular Atrophy in 7MM is expected to change from 2019-2028.

Spinal Muscular Atrophy Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.

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

Expected launch of therapies for Spinal Muscular Atrophy such as Zolgensama (Novartis), Risdiplam (Astellas Pharma/Cytokinetics), Branaplam (Novartis) and other targeted therapies in the forecast period [2019-2028] will also create a positive impact on the Spinal Muscular Atrophy market.

Spinal Muscular Atrophy Report Insights

Patient Population
Therapeutic Approaches
Market Size and Trends
Market Opportunities
Impact of upcoming Therapies

Spinal Muscular Atrophy Report Key Strengths

10 Year Forecast
7MM Coverage
Epidemiology Segmentation
Drugs Uptake
Highly Analyzed Market
Key Cross Competition

Spinal Muscular Atrophy Report Assessment

Current Treatment Practices
Unmet Needs
Market Attractiveness
Market Drivers and Barriers

Key Benefits

This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving Spinal Muscular Atrophy market
Organize sales and marketing efforts by identifying the best opportunities for Spinal Muscular Atrophy market
To understand the future market competition in the Spinal Muscular Atrophy market.

1. Key Insights

2. Spinal Muscular Atrophy Market Overview at a Glance

2.1. Market Share (%) Distribution of Spinal Muscular Atrophy in 2017
2.2. Market Share (%) Distribution of Spinal Muscular Atrophy in 2028

3. Disease Background and Overview: Spinal Muscular Atrophy (SMA)

3.1. Introduction
3.2. Classification
3.3. Causes
3.4. Pathophysiology
3.5. Biomarkers in Spinal Muscular Atrophy
3.6. Symptoms
3.7. Diagnosis
3.8. Differential Diagnosis
3.9. Diagnostic Algorithm

4. Epidemiology and Patient Population

4.1. Key Findings
4.2. 7MM Total Patient Population of Spinal Muscular Atrophy
4.3. 7MM Total Diagnosed Patient Population of Spinal Muscular Atrophy

5. Country Wise-Epidemiology of Spinal Muscular Atrophy

5.1. United States

5.1.1. Assumptions and Rationale
5.1.2. Spinal Muscular Atrophy Prevalent Cases in the United States
5.1.3. Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United States
5.1.4. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United States
5.1.5. Spinal Muscular Atrophy Mutation specific Prevalence in the United States

5.2. EU5 Countries

5.2.1. Assumptions and Rationale

5.3. Germany

5.3.1. Spinal Muscular Atrophy Prevalent Cases in Germany
5.3.2. Spinal Muscular Atrophy Diagnosed Prevalent Cases in Germany
5.3.3. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Germany
5.3.4. Spinal Muscular Atrophy Mutation specific Prevalence in Germany

5.4. France

5.4.1. Spinal Muscular Atrophy Prevalent Cases in France
5.4.2. Spinal Muscular Atrophy Diagnosed Prevalent Cases in France
5.4.3. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in France
5.4.4. Spinal Muscular Atrophy Mutation specific Prevalence in France

5.5. Italy

5.5.1. Spinal Muscular Atrophy Prevalent Cases in Italy
5.5.2. Spinal Muscular Atrophy Diagnosed Prevalent Cases in Italy
5.5.3. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Italy
5.5.4. Spinal Muscular Atrophy Mutation specific Prevalence in Italy

5.6. Spain

5.6.1. Spinal Muscular Atrophy Prevalent Cases in Spain
5.6.2. Spinal Muscular Atrophy Diagnosed Prevalent Cases in Spain
5.6.3. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Spain
5.6.4. Spinal Muscular Atrophy Mutation specific Prevalence in Spain

5.7. United Kingdom

5.7.1. Spinal Muscular Atrophy Prevalent Cases in the United Kingdom
5.7.2. Spinal Muscular Atrophy Diagnosed Prevalent Cases in the United Kingdom
5.7.3. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in the United Kingdom
5.7.4. Spinal Muscular Atrophy Mutation specific Prevalence in the United Kingdom

5.8. Japan

5.8.1. Assumptions and Rationale
5.8.2. Spinal Muscular Atrophy Prevalent Cases in Japan
5.8.3. Spinal Muscular Atrophy Diagnosed Prevalent Cases in Japan
5.8.4. Spinal Muscular Atrophy Type specific Diagnosed Prevalence in Japan
5.8.5. Spinal Muscular Atrophy Mutation specific Prevalence in Japan

6. Treatment

6.1. Approaches for Spinal Muscular Atrophy

6.1.1. Nutrition
6.1.2. Respiratory Care Assessment
6.1.3. Respiratory muscle weakness
6.1.4. Orthopedic Care
6.1.5. Swallowing muscle weakness
6.1.6. Rehabilitation
6.1.7. Medications
6.1.8. Acute care management
6.1.9. Medical care site/hospital capacity considerations

6.2. Potential Therapeutic Strategies

7. Treatment Algorithm

7.1. American Academy of Neurology Guidelines

7.1.1. Treatment Algorithm for Infants Diagnosed with Spinal Muscular Atrophy through Newborn Screening
7.1.2. Guidelines for follow up of follow up of infants identified as having SMA with four copies of SMN2

8. United States Association Current Recommendations for Spinal Muscular Atrophy (Guidelines)

9. Europe Association Current Recommendations for Spinal Muscular Atrophy

10. Japan Association Current Recommendations for Spinal Muscular Atrophy

11. Unmet Needs

12. Marketed Drugs

12.1. Spinraza (nusinersen): Biogen

12.1.1. Drug Description
12.1.2. Mechanism of Action
12.1.3. Regulatory Milestones
12.1.4. Other Development Activities
12.1.5. Safety and Efficacy
12.1.6. Advantages and Disadvantages
12.1.7. Product Profile

13. Emerging Drugs

13.1. Key Cross Competition
13.2. Zolgensma: Novartis

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. Risdiplam: Hoffmann-La Roche

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. Reldesemtiv: Astellas Pharma/Cytokinetics

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

13.5. Branaplam: Novartis

13.5.1. Product Description
13.5.2. Other Development Activities
13.5.3. Clinical Development
13.5.4. Safety and Efficacy
13.5.5. Product Profile

13.6. Amifampridine Phosphate: Catalyst Pharmaceuticals

13.6.1. Product Description
13.6.2. Clinical Development

13.7. BVS 857: Novartis

13.7.1. Product Description
13.7.2. Clinical Development
13.7.3. Safety and Efficacy
13.7.4. Product Profile

14. Spinal Muscular Atrophy: 7 Major Market Analysis

14.1. Key Findings
14.2. Market Size of Spinal Muscular Atrophy in 7MM

15. The United States Market Outlook

15.1. United States Market Size

15.1.1. Total Market size of Spinal Muscular Atrophy
15.1.2. Market Size by Therapies

16. EU-5 Countries: Market Outlook

16.1. Germany

16.1.1. Total Market size of Spinal Muscular Atrophy in Germany
16.1.2. Market Size by Therapies

16.2. France

16.2.1. Total Market Size of Spinal Muscular Atrophy
16.2.2. Market Size by Therapies

16.3. Italy

16.3.1. Total Market Size of Spinal Muscular Atrophy
16.3.2. Market Size by Therapies

16.4. Spain

16.4.1. Total Market Size of Spinal Muscular Atrophy
16.4.2. Market Size by Therapies

16.5. United Kingdom

16.5.1. Total Market Size of Spinal Muscular Atrophy
16.5.2. Market Size by Therapies

17. Japan: Market Outlook

17.1. Japan market Size

17.1.1. Total Market Size of Spinal Muscular Atrophy
17.1.2. Market Size by Therapies

18. Market Drivers

19. Market Barriers

20. Appendix

20.1. Report Methodology

脊髄性筋萎縮症（SMA）- 市場考察、疫学、市場予測

Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast - 2028

目次

第1章 レポートの概要

第2章 脊髄性筋萎縮症（SMA）市場の概要

第3章 脊髄性筋萎縮症（SMA）：背景と概要

第4章 疫学と患者人口

第5章 治療のガイドライン

第6章 アンメットニーズ

第7章 上市済みの治療薬

第8章 多元的競争

第9章 開発中の新薬

第10章 その他の新治療法

第11章 市場規模

第12章 米国

第13章 欧州

第14章 市場の成長要因と障壁

第15章 付録

第16章 調査手法

第17章 コンサルティングサービス

第18章 免責事項