チミジンキナーゼ2欠乏症（TK2d）- 市場考察、疫学、市場予測（2034年）

主なハイライト

• チミジンキナーゼ2欠乏症（TK2d）は主に、TK2遺伝子の変異によりミトコンドリア機能に影響を及ぼす、生命を脅かすまれな遺伝性疾患です。この疾患はミトコンドリアDNA（mtDNA）の著しい減少を引き起こし、重篤な筋力低下やその他の全身合併症を引き起こします。
• TK2dは2001年に重度の筋疾患を有する4人の小児で初めて報告されました。報告されたすべての患者にある程度の筋力低下がみられるが、重症度、発症年齢、疾患の進行は人それぞれ異なります。
• 現在、TK2dを特異的に標的とする世界的に承認された治療法はなく、治療は主に集学的アプローチによる症状管理に重点が置かれています。
• 2023年、TK2dの市場シェアは米国が約60%ともっとも高く、欧州4ヶ国（ドイツ、スペイン、イタリア、フランス）、英国、日本がこれに続きます。
• 主要7市場における2023年の総市場規模は約100万米ドルです。
• TK2dは、症状、病歴、臨床検査、研究室・遺伝子検査によって診断されます。TK2遺伝子の変異に関する遺伝子検査で診断が確定します。クレアチンキナーゼ値の上昇や筋電図検査によるミオパチーの変化が診断の裏付けとなります。罹患者とその家族には遺伝カウンセリングが推奨されます。
• 2023年、米国は主要7市場におけるTK2d患者全体の約50%を占め、これは主要7市場の中でもっとも多いです。
• 2023年、米国は主要7市場でもっとも治療されたTK2d患者数が多く、約350人、次いでドイツの約90人でした。これらの数は予測期間に増加する見込みです。
• TK2dのパイプラインはそれほど強固ではありませんが、MT1621（Doxecitine and Doxribtimine）という可能性のある薬剤が1つあります。
• 2022年1月、UCBとZogenixは、UCBがZogenixを買収する正式契約を締結したと発表しました。

チミジンキナーゼ2欠乏症（TK2d）市場の見通し

TK2dに特化したFDA承認薬はありませんが、治療の中心は主にデオキシヌクレオシド療法であり、ミトコンドリアDNAの複製と維持に必要な基礎的要素を提供します。この治療法は臨床試験で有望視されており、最小の副作用で筋力と呼吸機能の改善を示しています。TK2遺伝子の突然変異による酵素欠乏をバイパスすることで、デオキシヌクレオシド療法は、この困難な疾患のよりよい管理に希望を与えるものです。進行中の研究では、患者の転帰とQOLをさらに向上させるためのさらなる治療戦略が模索され続けています。

一言で言えば、チミジンキナーゼ2欠乏症の治療法として検討されているものは多くありません。予測期間（2024年～2034年）に市場に参入する上記の有望な候補についてコメントするのは時期尚早です。最終的には、この薬剤は今後数年間で、チミジンキナーゼ2欠乏症の情勢に大きな変化をもたらすと見られます。この治療領域は、世界の医療費の改善により、今後数年間で大きくプラスに転じると予測されます。

当レポートでは、チミジンキナーゼ2欠乏症（TK2d）の主要7市場（米国、ドイツ、スペイン、イタリア、フランス、英国、日本）について調査分析し、各地域の市場規模、現在の治療法、アンメットニーズ、新薬などの情報を提供しています。

目次

第1章 重要考察

第2章 レポートのイントロダクション

第3章 TK2d市場の概要

• 主要7市場のTK2dの市場シェアの分布（2023年）
• 主要7市場のTK2dの市場シェアの分布（2034年）

第4章 調査手法

第5章 エグゼクティブサマリー

第6章 疾患の背景と概要

• PMM
• PMMの種類
  • KSS
  • MELAS
  • MERRF
  • CPEO
  • CoQ10欠乏症
  • TK2d
• TK2dのサブタイプ
  • 早期発症型（1歳以下）
  • 小児期発症型（1歳～12歳）
  • 遅発型（12歳超）
• TK2dの症状
• TK2dの危険因子
• TK2dの病因
• TK2dの診断
  • 検査所見
  • 神経イメージング
  • 筋生検所見
  • TK2dの分子遺伝学
  • TK2dのバイオマーカー
  • 診断アルゴリズム
  • 鑑別診断

第7章 TK2dの治療と管理

• TK2dの治療と診断：CSURs-NMDとスペインの関連研究団体の役割
• 治療アルゴリズム

第8章 疫学と患者人口

• 主な調査結果
• 前提条件と根拠
• 主要7市場のTK2dの総患者数
• 主要7市場のTK2dの治療された総患者数
• 米国
• 欧州4ヶ国・英国
• 日本

第9章 ペイシェントジャーニー

第10章 新治療法

• MT1621（Doxecitine and Doxribtimine）：UCB Biosciences

第11章 市場の分析

• 主な調査結果
• 市場見通し
• 主な市場予測の前提条件
• 主要7市場のTK2dの総市場規模
• 主要7市場のTK2dの総市場規模：治療法別
• 米国の市場規模
• 欧州4ヶ国・英国の市場規模
• 日本の市場規模

第12章 KOLの見解

第13章 SWOT分析

第14章 アンメットニーズ

第15章 市場参入と償還

• 米国
• 欧州4ヶ国・英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本

第16章 付録

第17章 DelveInsightのサービス内容

第18章 免責事項

List of Tables

• Table 1: Summary of TK2d Market and Epidemiology (2020-2034)
• Table 2: Primary Mitochondrial Classical Syndromes With Muscle Involvement
• Table 3: Clinical Manifestations and Muscle Histological Features of TK2d Subtypes
• Table 4: Differential Diagnosis of TK2d Subtypes
• Table 5: Total Prevalent Cases of TK2d in 7MM (2020-2034)
• Table 6: Total Treated Cases of TK2d in 7MM (2020-2034)
• Table 7: Total Prevalent Cases of TK2d in the United States (2020-2034)
• Table 8: Total Diagnosed Prevalent Cases of TK2d in the United States (2020-2034)
• Table 9: Gender-specific Cases of TK2d in the United States (2020-2034)
• Table 10: Phenotype-specific Cases of TK2d in the United States (2020-2034)
• Table 11: Total Treated Cases of TK2d in the United States (2020-2034)
• Table 12: Total Prevalent Cases of TK2d in EU4 and the UK (2020-2034)
• Table 13: Total Diagnosed Prevalent Cases of TK2d in EU4 and the UK (2020-2034)
• Table 14: Gender-specific cases of TK2d in EU4 and the UK (2020-2034)
• Table 15: Severity-specific Cases of TK2d in EU4 and the UK (2020-2034)
• Table 16: Total Treated Cases of TK2d in 7MM (2020-2034)
• Table 17: Total Diagnosed Prevalent Cases of TK2d in Japan (2020-2034)
• Table 18: Total Diagnosed Prevalent Cases of TK2d in Japan (2020-2034)
• Table 19: Gender-specific Cases of TK2d in Japan (2020-2034)
• Table 20: Severity-specific Cases of TK2d in Japan (2020-2034)
• Table 21: Total Treated Cases of TK2d in Japan (2020-2034)
• Table 22: MT1621 (Doxecitine and Doxribtimine) Clinical Trial Description, 2024
• Table 23: Key Market Forecast Assumption of TK2d in the United States
• Table 24: Key Market Forecast Assumption of TK2d in EU4 and the UK
• Table 25: Key Market Forecast Assumption of TK2d in Japan
• Table 26: Total Market Size of TK2d in the 7MM, in USD million (2020-2034)
• Table 27: Market Size of TK2d by Therapies in the 7MM, in USD million (2020-2034)
• Table 28: Total Market Size of TK2d in the United States, in USD million (2020-2034)
• Table 29: Market Size of TK2d by Therapies in the United States in USD million (2020-2034)
• Table 30: Total Market Size of TK2d in EU4 and the UK, in USD million (2020-2034)
• Table 31: Market Size of TK2d by Therapies in EU4 and the UK in USD million (2020-2034)
• Table 32: Total Market Size of TK2d in Japan, in USD million (2020-2034)
• Table 33: Market Size of TK2d by Therapies in Japan in USD million (2020-2034)

List of Figures

• Figure 1: Most Common Symptoms
• Figure 2: Representation of Multiple Effectors Involved in the Pathogenesis of Mitochondrial Dysfunction.
• Figure 3: Representation of the Role of Mitochondrial Dysfunction and Disrupted Bioenergetics
• Figure 4: TK2 Gene Variants Distribution
• Figure 5: Proposed TK2d Diagnostic Algorithm
• Figure 6: Using Technology as Part of a Holistic Approach to Disease Management in TK2d
• Figure 7: Pharmacological Mechanism of Nucleoside Therapy in TK2d
• Figure 8: Key Milestones in TK2d Diagnostics and Treatment: Role of the Spanish Research Affiliates and Collaborators
• Figure 9: Treatment Algorithm for TK2d
• Figure 10: Total Prevalent Cases of TK2d in 7MM (2020-2034)
• Figure 11: Total Treated Cases of TK2d in 7MM (2020-2034)
• Figure 12: Total Prevalent Cases of TK2d in the United States (2020-2034)
• Figure 13: Total Diagnosed Prevalent Cases of TK2d in the United States (2020-2034)
• Figure 14: Gender-specific cases of TK2d in the United States (2020-2034)
• Figure 15: Phenotype-specific Cases of TK2d in the United States (2020-2034)
• Figure 16: Total Treated Cases of TK2d in the United States (2020-2034)
• Figure 17: Total Prevalent Cases of TK2d in EU4 and the UK (2020-2034)
• Figure 18: Total Diagnosed Prevalent Cases of TK2d in EU4 and the UK (2020-2034)
• Figure 19: Gender-specific Cases of TK2d in EU4 and the UK (2020-2034)
• Figure 20: Severity-specific Cases of TK2d in EU4 and the UK (2020-2034)
• Figure 21: Total Treated Cases of TK2d in EU4 and the UK (2020-2034)
• Figure 22: Total Prevalent Cases of TK2d in Japan (2020-2034)
• Figure 23: Total Diagnosed Prevalent Cases of TK2d in Japan (2020-2034)
• Figure 24: Gender-specific cases of TK2d in Japan (2020-2034)
• Figure 25: Severity-specific Cases of TK2d in Japan (2020-2034)
• Figure 26: Total Treated Cases of TK2d in Japan (2020-2034)
• Figure 27: Total Market Size of TK2d in the 7MM (2020-2034)
• Figure 28: Market Size of TK2d by Therapies in the 7MM (2020-2034)
• Figure 29: Total Market Size of TK2d in the US (2020-2034)
• Figure 30: Market Size of TK2d by Therapies in the United States (2020-2034)
• Figure 31: Total Market Size of TK2d in EU4 and the UK (2020-2034)
• Figure 32: Market Size of TK2d by Therapies in EU4 and the UK (2020-2034)
• Figure 33: Total Market Size of TK2d in Japan (2020-2034)
• Figure 34: Market Size of TK2d by Therapies in Japan (2020-2034)
• Figure 35: Health Technology Assessment
• Figure 36: Reimbursement Process in Germany
• Figure 37: Reimbursement Process in France
• Figure 38: Reimbursement Process in Italy
• Figure 39: Reimbursement Process in Spain
• Figure 40: Reimbursement Process in the United Kingdom
• Figure 41:Reimbursement Process in Japan

Key Highlights:

• Thymidine Kinase 2 deficiency (TK2d) is a rare, life-threatening genetic disorder that primarily affects mitochondrial function due to mutations in the TK2 gene. This condition leads to a significant reduction in mitochondrial DNA (mtDNA), resulting in severe muscle weakness and other systemic complications.
• TK2d was first described in 2001 in four children with severe muscle disease. All patients described have some degree of muscle weakness, but the severity, age of onset, and disease progression vary from person to person.
• Currently, there are no globally approved therapies that specifically target TK2d, leaving treatment primarily focused on symptom management through a multidisciplinary approach.
• In 2023, the United States holds the largest market share for TK2d, accounting for approximately 60%, compared to the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.
• The total market size in the 7MM was approximately USD 1 million in 2023.
• TK2d is diagnosed through symptoms, patient history, clinical exam, and laboratory and genetic tests. Genetic testing for mutations in the TK2 gene confirms the diagnosis. Elevated creatine kinase levels and electromyography showing myopathic changes can support the diagnosis. Genetic counseling is recommended for affected individuals and their families.
• In 2023, the United States represented around 50% of the total prevalent cases of TK2d in the 7MM, the highest among them.
• In 2023, the United States had the highest number of treated TK2d cases in the 7MM, with approximately 350 cases, followed by Germany with around 90 cases. These numbers are expected to rise during the forecast period.
• TK2d pipeline is not so robust but possesses one potential drug i.e. MT1621 (Doxecitine and Doxribtimine).
• In January 2022, UCB and Zogenix announced that the companies had entered into a definitive agreement under which UCB acquired Zogenix.

Report Summary

• The report offers extensive knowledge regarding the epidemiology segments and predictions, presenting a deep understanding of the potential future growth in diagnosis rates, disease progression, and treatment guidelines. It provides comprehensive insights into these aspects, enabling a thorough assessment of the subject matter.
• Additionally, an all-inclusive account of the current management techniques and emerging therapies and the elaborative profiles of late-stage (Phase II and Phase I) and prominent therapies that would impact the current treatment landscape and result in an overall market shift has been provided in the report.
• The report also encompasses a comprehensive analysis of the Thymidine Kinase 2 deficiency market, providing an in-depth examination of its historical and projected market size (2020-2034). It also includes the market share of therapies, detailed assumptions, and the underlying rationale for our methodology. The report also includes drug outreach coverage in the 7MM region.
• The report includes qualitative insights that provide an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, including experts from various hospitals and prominent universities, patient journey, and treatment preferences that help shape and drive the 7MM Thymidine Kinase 2 deficiency market.

Market

A few key players are leading the treatment landscape of Thymidine Kinase 2 deficiency, such as UCB, and others. The details of the country-wise and therapy-wise market size have been provided below.

• In the total market size of Thymidine Kinase 2 deficiency in the 7MM, the United States accounted for the highest market share, i.e. approximately 60% in 2023, followed by Germany.
• Among EU4 and the UK, Germany accounted for almost 9% of the market size in 2023.
• The United States accounted for approximately USD 0.5 million in 2023.
• Currently, only Standard of Care (SOC) is being used as the only option of therapy for the management of Thymidine Kinase 2 deficiency.

Thymidine Kinase 2 deficiency (TK2d) Drug Chapters

The section dedicated to drugs in the Thymidine Kinase 2 deficiency report provides an in-depth evaluation of late-stage pipeline drugs (Phase II) related to Thymidine Kinase 2 deficiency. The drug chapters section provides valuable information on various aspects related to clinical trials of Thymidine Kinase 2 deficiency, such as the pharmacological mechanisms of the drugs involved, designations, approval status, patent information, and a comprehensive analysis of the pros and cons associated with each drug. Furthermore, it presents the most recent news updates and press releases on drugs targeting Thymidine Kinase 2 deficiency.

Emerging Therapies

MT1621 (Doxecitine and Doxribtimine): UCB Biosciences

Doxecitine and doxribtimine (MT1621) is a fixed-dose combination therapy that targets the underlying pathophysiology of Thymidine Kinase 2 deficiency (TK2d) by restoring mitochondrial DNA (mtDNA) replication fidelity. Doxecitine and doxribtimine consist of a combination of deoxynucleosides (the building blocks of mtDNA) given orally. Deoxynucleoside combination therapy improves nucleotide balance, increases mtDNA copy number, improves cell function, and prolongs life in preclinical models of TK2d. By increasing the levels of thymidine and deoxycytidine in the body, the medicine is expected to make up for the deficiencies in TK2 activity, thereby improving the production of mitochondrial DNA and helping relieve the patient's symptoms.

Doxecitine and doxribtimine are in clinical development for the treatment of TK2d. In the pivotal phase II trial (NCT03845712), doxecitine and doxribtimine are administered orally up to a maximum of 800 mg/kg/day (400 mg/kg/day of dC and 400 mg/kg/day of dT) as tolerated. In February 2019, the FDA granted Breakthrough Therapy designation, MT1621 has also been granted PRIME designation by the EMA and Orphan Drug Designation (ODD) by both the FDA and EMA in 2018.

Thymidine Kinase 2 deficiency (TK2d) Market Outlook

Although there are no FDA-approved drugs specifically for TK2d, treatment primarily centers on deoxynucleoside therapy, which provides the essential building blocks needed for mitochondrial DNA replication and maintenance. This therapy has shown promise in clinical trials, demonstrating improvements in muscle strength and respiratory function with minimal side effects. By bypassing the enzymatic deficiency caused by mutations in the TK2 gene, deoxynucleoside therapy offers hope for better management of this challenging condition. Ongoing research continues to explore additional therapeutic strategies to further enhance patient outcomes and quality of life.

In a nutshell, not many potential therapies are being investigated to manage Thymidine Kinase 2 deficiency. Even though it is too soon to comment on the above-mentioned promising candidate to enter the market during the forecast period (2024-2034). Eventually, this drug will create a significant difference in the landscape of Thymidine Kinase 2 deficiency in the coming years. The treatment space is expected to experience a significant positive shift in the coming years owing to the improvement in healthcare spending worldwide.

Thymidine Kinase 2 deficiency (TK2d) Disease Understanding and Treatment

Thymidine Kinase 2 deficiency (TK2d) Overview

Thymidine Kinase 2 deficiency is a rare autosomal recessive disorder caused by mutations in the TK2 gene, which is crucial for mitochondrial DNA (mtDNA) maintenance. This condition leads to a significant reduction in mtDNA, resulting in progressive muscle weakness and various systemic complications. Patients often present with symptoms such as weakness in the limbs, respiratory difficulties, and issues with eye movement and swallowing. The severity and progression of the disease can vary widely among individuals, with some experiencing early onset and rapid decline, while others may have a later onset with a slower progression.

Thymidine Kinase 2 deficiency (TK2d) Diagnosis

Diagnosis of TK2d typically involves a combination of genetic testing and clinical evaluations. Genetic testing is conducted to identify mutations in the TK2 gene, confirming the diagnosis. In addition to genetic analysis, clinicians may perform muscle biopsies to assess for signs of mitochondrial myopathies, such as ragged red fibers or abnormal mitochondria under microscopic examination. The comprehensive evaluation of symptoms alongside these diagnostic methods helps to establish a clear diagnosis and differentiate TK2d from other mitochondrial disorders.

Thymidine Kinase 2 deficiency (TK2d) Treatment

Currently, there are no globally approved therapies that specifically target TK2d, leaving treatment primarily focused on symptom management through a multidisciplinary approach. This care strategy aims to alleviate complications and enhance the quality of life for affected individuals. Neurological support often involves tailored physical and occupational therapy to address progressive muscle weakness and improve mobility. For patients experiencing respiratory insufficiency, non-invasive or invasive ventilatory support is crucial to maintain adequate oxygenation and prevent respiratory failure. Nutritional management is another critical aspect, as high-calorie diets and, in severe cases, gastrostomy feeding are often required to counter malnutrition and ensure sufficient energy intake. Additionally, mobility aids such as wheelchairs are frequently necessary for individuals with advanced muscle weakness, enabling greater independence and reducing the burden of physical limitations.

Thymidine Kinase 2 deficiency (TK2d) Epidemiology

The Thymidine Kinase 2 deficiency epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Cases, Total Diagnosed Prevalent Cases, Gender-specific Cases, Phenotype-specific Cases, and Treated Cases of Thymidine Kinase 2 deficiency in the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• Among the 7MM, the United States accounted for the highest number of cases of Thymidine Kinase 2 deficiency in 2023, with nearly 560 cases. These cases are anticipated to increase by 2034.
• In 2023, the United States reported the highest number of infantile-onset myopathy cases of TK2d, with ~150 cases, followed by childhood-onset myopathy and late-onset myopathy cases, with ~140 and ~60 cases, respectively.
• In 2023, Gender-specific cases of TK2d in the United States were ~160 and ~200 for women and men, respectively.
• Among EU4 and the UK, Germany accounted for the highest number of total prevalent cases in 2023, with approximately 150 cases.

KOL Views

To stay abreast of the latest trends in the market, we conduct primary research by seeking the opinions of Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) who work in the relevant field. This helps us fill any gaps in data and validate our secondary research.

We have reached out to industry experts to gather insights on various aspects of Thymidine Kinase 2 deficiency, including the evolving treatment landscape, patients' reliance on conventional therapies, their acceptance of therapy switching, drug uptake, and challenges related to accessibility. The experts we contacted included medical/scientific writers, professors, and researchers from prestigious universities in the US, Europe, the UK, and Japan.

Our team of analysts at Delveinsight connected with more than 10 KOLs across the 7MM. We contacted institutions such as the Meyer Hospital, Instituto de Biomedicina de Sevilla, Centre for Biomedical Network Research on Rare Diseases (CIBERER), etc., among others. By obtaining the opinions of these experts, we gained a better understanding of the current and emerging treatment patterns in the Thymidine Kinase 2 deficiency market, which will assist our clients in analyzing the overall epidemiology and market scenario.

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 disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, designation, 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 trials for Thymidine Kinase 2 deficiency, one of the most important primary endpoints was achieving the number of participants who experience Adverse Events (AEs), etc. Based on these, the overall efficacy is evaluated.

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 route of administration, order of entry and designation, 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

Because newly authorized drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved 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.

Thymidine Kinase 2 deficiency (TK2d) Report Insights

• Patient Population
• Therapeutic Approaches
• Thymidine Kinase 2 deficiency Market Size and Trends
• Existing Market Opportunity

Thymidine Kinase 2 deficiency (TK2d) Report Key Strengths

• Eleven-year Forecast
• The 7MM Coverage
• Thymidine Kinase 2 deficiency Epidemiology Segmentation
• Key Cross Competition

Thymidine Kinase 2 deficiency (TK2d) Report Assessment

• Current Treatment Practices
• Reimbursements
• Market Attractiveness
• Qualitative Analysis (SWOT, Conjoint Analysis, Unmet needs)

Key Questions:

• Would there be any changes observed in the current treatment approach?
• Will there be any improvements in Thymidine Kinase 2 deficiency management recommendations?
• Would research and development advances pave the way for future tests and therapies for Thymidine Kinase 2 deficiency?
• Would the diagnostic testing space experience a significant impact and lead to a positive shift in the treatment landscape of Thymidine Kinase 2 deficiency?
• What kind of uptake will the new therapies witness in the coming years in Thymidine Kinase 2 deficiency patients?

Table of Contents

1. Key Insights

2. Report Introduction

3. TK2d Market Overview at a Glance

• 3.1. Market Share (%) Distribution of TK2d in 2023 in the 7MM
• 3.2. Market Share (%) Distribution of TK2d in 2034 in the 7MM

4. Methodology

5. Executive Summary

6. Disease Background and Overview

• 6.1. PMM
• 6.2. Types of PMM
  • 6.2.1. KSS
  • 6.2.2. MELAS
  • 6.2.3. MERRF
  • 6.2.4. CPEO
  • 6.2.5. CoQ10 deficiencies
  • 6.2.6. TK2d
• 6.3. Subtypes of TK2d
  • 6.3.1. Early Onset Form (=1-Year Old)
  • 6.3.2. Childhood Onset Form (> 1 to =12 Years Old)
  • 6.3.3. Late Onset Form (>12 Years Old)
• 6.4. Symptoms of TK2d
• 6.5. Risk Factors 0f TK2d
• 6.6. Pathogenesis of TK2d
• 6.7. Diagnosis of TK2d
  • 6.7.1. Laboratory Findings
  • 6.7.2. Neuroimaging
  • 6.7.3. Muscle Biopsy Findings
  • 6.7.4. Molecular Genetics of TK2d
  • 6.7.5. Biomarkers in TK2d
  • 6.7.6. Diagnostic Algorithm
  • 6.7.7. Differential Diagnosis

7. Treatment and Management of TK2d

• 7.1. TK2d Treatment and Diagnosis: Role of CSURs-NMD and Spanish Research Affiliates
• 7.2. Treatment Algorithm

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale
• 8.3. Total Prevalent Cases of TK2d in 7MM
• 8.4. Total Treated Cases of TK2d in 7MM
• 8.5. The United States
  • 8.5.1. Total Prevalent Cases of TK2d in the United States
  • 8.5.2. Total Diagnosed Prevalent Cases of TK2d in the United States
  • 8.5.3. Gender-specific Cases of TK2d in the United States
  • 8.5.4. Phenotype-specific Cases of TK2d in the United States
  • 8.5.5. Treated Cases of TK2d in the United States
• 8.6. EU4 and the UK
  • 8.6.1. Total Prevalent Cases of TK2d in EU4 and the UK
  • 8.6.2. Total Diagnosed Prevalent Cases of TK2d in EU4 and the UK
  • 8.6.3. Gender-specific Cases of TK2d in EU4 and the UK
  • 8.6.4. Phenotype-specific Cases of TK2d in EU4 and the UK
  • 8.6.5. Total Treated Cases of TK2d in EU4 and the UK
• 8.7. Japan
  • 8.7.1. Total Prevalent Cases of TK2d Japan
  • 8.7.2. Total Diagnosed Prevalent Cases of TK2d Japan
  • 8.7.3. Gender-specific Cases of TK2d in Japan
  • 8.7.4. Phenotype-specific Cases of TK2d in Japan
  • 8.7.5. Treated Cases of TK2d in Japan

9. Patient Journey

10. Emerging Therapies

• 10.1. MT1621 (Doxecitine and Doxribtimine): UCB Biosciences
  • 10.1.1. Product Description
  • 10.1.2. Other Developmental Activities
  • 10.1.3. Clinical Developmental Activities
  • 10.1.4. Safety and Efficacy
  • 10.1.5. Analyst View

11. Market Analysis

• 11.1. Key Findings
• 11.2. Market Outlook
• 11.3. Key Market Forecast Assumptions
• 11.4. Total Market Size of TK2d in the 7MM
• 11.5. Total Market Size of TK2d by Therapies in the 7MM
• 11.6. United States Market Size
  • 11.6.1. Total Market Size of TK2d in the US (2020-2034)
  • 11.6.2. Market Size of TK2d by Therapies in the United States (2020-2034)
• 11.7. EU4 and the UK Market Size
  • 11.7.1. Total Market Size of TK2d in EU4 and the UK (2020-2034)
  • 11.7.2. Market Size of TK2d by Therapies in EU4 and the UK (2020-2034)
• 11.8. Japan Market Size
  • 11.8.1. Total Market Size of TK2d in Japan (2020-2034)
  • 11.8.2. Market Size of TK2d by Therapies (2020-2034)

12. KOL Views

13. SWOT Analysis

14. Unmet Needs

15. Market Access and Reimbursement

• 15.1. United States
  • 15.1.1. Centre for Medicare and Medicaid Services (CMS)
• 15.2. EU4 and the UK
  • 15.2.1. Germany
  • 15.2.2. France
  • 15.2.3. Italy
  • 15.2.4. Spain
  • 15.2.5. United Kingdom
• 15.3. Japan
  • 15.3.1. MHLW

16. Appendix

• 16.1. Bibliography
• 16.2. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer