クラッベ病治療市場：疾患タイプ、治療法、流通チャネル別-2025～2030年の世界予測

クラッベ病治療市場は、2023年に17億2,000万米ドルと評価され、2024年には18億3,000万米ドルに達すると予測され、CAGR 6.49％で成長し、2030年には26億8,000万米ドルに達すると予測されています。

クラッベ病は、神経系に影響を及ぼすまれでしばしば致死的な遺伝子疾患であり、その複雑さと限られた治療選択肢のために、ますます医療研究の焦点になりつつあります。治療法としては、造血幹細胞移植（HSCT）、酵素補充療法、遺伝子治療が主に行われているが、これらの治療法の有効性や利用可能性が限られているため、市場ニーズは依然として高いです。このアンメットニーズとバイオテクノロジーや医薬品の進歩が相まって、より効果的で利用しやすい解決策の開発に関心が集まっています。主要成長要因としては、疾患に対する認識と診断の高まり、遺伝子研究の進歩、希少疾患を対象とした政府の支援ポリシーなどが挙げられます。最新のビジネス機会は、CRISPRのような遺伝子編集技術や新規薬剤製剤の進歩にあり、画期的な治療と患者のQOL向上の可能性を提供しています。とはいえ、研究開発コストの高さ、希少疾患に関連する規制の複雑さ、限られた患者プールなど、この市場は大きな課題に直面しています。こうした制約を克服するために、企業は共同研究イニシアティブや学術機関との提携に注力することができます。個別化医療における革新的な取り組み、特に遺伝子プロファイル別の治療の個別化は、潜在的な成長と差別化をもたらすエキサイティングなセグメントです。市場の性質は依然として課題的であると同時に有望でもあり、急速な技術進歩に加え、物流や資金面でのハードルが高いという特徴があります。市場への参入とリーダーシップを成功させるには、研究開発への戦略的投資、新たな規制要件への機敏な対応、重要なアンメット・ニーズに専門的に対応するためのペイシェント・ジャーニーに対する確かな理解が必要であると考えられます。技術動向、ポリシー転換、競合の動きをモニターするための継続的な市場調査は、クラッベ病治療市場でのポジショニングと持続的成長に不可欠です。

市場力学：急速に進化するクラッベ病治療市場の主要市場洞察を公開

クラッベ病治療市場は、需要と供給のダイナミックな相互作用によって変貌を遂げています。このような市場力学の進化を理解することで、企業は十分な情報に基づいた投資決定、戦略的意思決定、新たなビジネス機会の獲得を行うことができます。これらの動向を包括的に把握することで、企業は政治的、地理的、技術的、社会的、経済的な領域にわたる様々なリスクを軽減することができるとともに、消費者行動とそれが製造コストや購買動向に与える影響をより明確に理解することができます。

• 市場促進要因
  • 世界のクラッベ病有病率の増加
  • 遺伝子治療と幹細胞移植研究の増加
  • 遺伝性疾患に対する認識を高めるための政府と民間の取り組み
• 市場抑制要因
  • クラッベ病の治療に伴う高コスト
• 市場機会
  • クラッベ病の診断と治療の進歩
  • クラッベ病の研究拡大のための有利な資金状況
• 市場課題
  • クラッベ病の検査と治療に関する倫理的問題

ポーターのファイブフォース：クラッベ病治療市場をナビゲートする戦略ツール

ポーターのファイブフォースフレームワークは、市場情勢の競合情勢を理解するための重要なツールです。ポーターのファイブフォースフレームワークは、企業の競合を評価し、戦略的機会を探るための明確な手法を記載しています。このフレームワークは、企業が市場内の勢力図を評価し、新規事業の収益性を判断するのに役立ちます。これら洞察により、企業は自社の強みを活かし、弱みに対処し、潜在的な課題を回避することができ、より強靭な市場でのポジショニングを確保することができます。

PESTLE分析：クラッベ病治療市場における外部からの影響の把握

外部マクロ環境要因は、クラッベ病治療市場の業績力学を形成する上で極めて重要な役割を果たします。政治的、経済的、社会的、技術的、法的、環境的要因の分析は、これらの影響をナビゲートするために必要な情報を記載しています。PESTLE要因を調査することで、企業は潜在的なリスクと機会をよりよく理解することができます。この分析により、企業は規制、消費者の嗜好、経済動向の変化を予測し、先を見越した積極的な意思決定を行う準備ができます。

市場シェア分析：クラッベ病治療市場における競合情勢の把握

クラッベ病治療市場の詳細な市場シェア分析により、ベンダーの業績を包括的に評価することができます。企業は、収益、顧客ベース、成長率などの主要指標を比較することで、競争上のポジショニングを明らかにすることができます。この分析により、市場の集中、セグメント化、統合の動向が明らかになり、ベンダーは競争が激化する中で自社の地位を高める戦略的な意思決定を行うために必要な知見を得ることができます。

FPNVポジショニングマトリックス：クラッベ病治療市場におけるベンダーのパフォーマンス評価

FPNVポジショニングマトリックスは、クラッベ病治療市場においてベンダーを評価するための重要なツールです。このマトリックスにより、ビジネス組織はベンダーのビジネス戦略と製品満足度に基づき評価することで、目標に沿った十分な情報に基づいた意思決定を行うことができます。4つの象限によりベンダーを明確かつ的確にセグメント化し、戦略目標に最適なパートナーやソリューションを特定することができます。

戦略分析と推奨：クラッベ病治療市場における成功への道筋を描く

クラッベ病治療市場の戦略分析は、世界市場でのプレゼンス強化を目指す企業にとって不可欠です。主要なリソース、能力、業績指標を検討することで、企業は成長機会を特定し、改善に取り組むことができます。このアプローチにより、競合情勢における課題を克服し、新たなビジネス機会を活かして長期的な成功を収めるための体制を整えることができます。

本レポートでは、主要な注目セグメントを網羅した市場の包括的な分析を提供しています。

1.市場の浸透度：現在の市場環境の詳細なレビュー、主要企業による広範なデータ、市場でのリーチと全体的な影響力を評価します。

2.市場の開拓度：新興市場における成長機会を特定し、既存セグメントにおける拡大可能性を評価し、将来の成長に向けた戦略的ロードマップを記載しています。

3.市場の多様化：最近の製品発売、未開拓の地域、産業の主要な進歩、市場を形成する戦略的投資を分析します。

4.競合の評価と情報：競合情勢を徹底的に分析し、市場シェア、事業戦略、製品ポートフォリオ、認証、規制当局の承認、特許動向、主要企業の技術進歩などを検証します。

5.製品開発とイノベーション：将来の市場成長を促進すると期待される最先端技術、研究開発活動、製品イノベーションをハイライトしています。

また、利害関係者が十分な情報に基づいた意思決定を行う上で役立つ重要な質問にも回答しています。

1.現在の市場規模と今後の成長予測は？

2.最高の投資機会を提供する製品、地域はどこか？

3.市場を形成する主要技術動向と規制の影響とは？

4.主要ベンダーの市場シェアと競合ポジションは？

5.ベンダーの市場参入・撤退戦略の原動力となる収益源と戦略的機会は何か？

目次

第1章 序文

第2章 調査手法

第3章 エグゼクティブサマリー

第4章 市場概要

第5章 市場洞察

• 市場力学
  • 促進要因
    • 世界中でクラッベ病の罹患率が増加
    • 遺伝子治療と幹細胞移植調査の増加
    • 遺伝性疾患に関する意識を高める政府と民間の取り組み
  • 抑制要因
    • クラッベ病の治療にかかる高額な費用
  • 機会
    • クラッベ病の診断と治療の進歩
    • クラッベ病の調査拡大に向けた好ましい資金調達情勢
  • 課題
    • クラッベ病の検査と治療に関する倫理的問題
• 市場セグメンテーション分析
• ポーターのファイブフォース分析
• PESTEL分析
  • 政治
  • 経済
  • 社会
  • 技術
  • 法律
  • 環境

第6章 クラッベ病治療市場：疾患タイプ別

• イントロダクション
• 幼児期
• 後発性

第7章 クラッベ病治療市場：治療別

• イントロダクション
• 抗けいれん薬
• 骨髄移植
• 筋弛緩薬

第8章 クラッベ病治療市場：流通チャネル別

• イントロダクション
• eコマース
• 病院薬局
• 小売薬局

第9章 南北アメリカのクラッベ病治療市場

• イントロダクション
• アルゼンチン
• ブラジル
• カナダ
• メキシコ
• 米国

第10章 アジア太平洋のクラッベ病治療市場

• イントロダクション
• オーストラリア
• 中国
• インド
• インドネシア
• 日本
• マレーシア
• フィリピン
• シンガポール
• 韓国
• 台湾
• タイ
• ベトナム

第11章 欧州・中東・アフリカのクラッベ病治療市場

• イントロダクション
• デンマーク
• エジプト
• フィンランド
• フランス
• ドイツ
• イスラエル
• イタリア
• オランダ
• ナイジェリア
• ノルウェー
• ポーランド
• カタール
• ロシア
• サウジアラビア
• 南アフリカ
• スペイン
• スウェーデン
• スイス
• トルコ
• アラブ首長国連邦
• 英国

第12章 競合情勢

• 市場シェア分析、2023年
• FPNVポジショニングマトリックス、2023年年
• 競合シナリオ分析
• 戦略分析と提言

企業一覧

• Abbott Laboratories
• Acorda Therapeutics Inc.
• Apteeus SAS
• Centogene N.V.
• Forge Biologics, Inc.
• Gain Therapeutics, Inc.
• GlaxoSmithKline Plc
• Johnson & Johnson
• M6P Therapeutics
• Neurogene Inc.
• Novartis AG
• Pfizer Inc.
• Polaryx Therapeutics, Inc
• Polpharma
• Sanofi S.A.
• Takeda Pharmaceutical Company Limited
• Teva Pharmaceutical Industries Ltd.
• UCB Pharmaceuticals

LIST OF FIGURES

• FIGURE 1. KRABBE DISEASE TREATMENT MARKET RESEARCH PROCESS
• FIGURE 2. KRABBE DISEASE TREATMENT MARKET SIZE, 2023 VS 2030
• FIGURE 3. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, 2018-2030 (USD MILLION)
• FIGURE 4. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY REGION, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 5. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 6. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2023 VS 2030 (%)
• FIGURE 7. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 8. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2023 VS 2030 (%)
• FIGURE 9. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 10. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2023 VS 2030 (%)
• FIGURE 11. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 12. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 13. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 14. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY STATE, 2023 VS 2030 (%)
• FIGURE 15. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY STATE, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 16. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 17. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 18. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2030 (%)
• FIGURE 19. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2023 VS 2024 VS 2030 (USD MILLION)
• FIGURE 20. KRABBE DISEASE TREATMENT MARKET SHARE, BY KEY PLAYER, 2023
• FIGURE 21. KRABBE DISEASE TREATMENT MARKET, FPNV POSITIONING MATRIX, 2023

LIST OF TABLES

• TABLE 1. KRABBE DISEASE TREATMENT MARKET SEGMENTATION & COVERAGE
• TABLE 2. UNITED STATES DOLLAR EXCHANGE RATE, 2018-2023
• TABLE 3. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, 2018-2030 (USD MILLION)
• TABLE 4. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 5. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 6. KRABBE DISEASE TREATMENT MARKET DYNAMICS
• TABLE 7. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 8. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY INFANTILE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 9. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY LATE-ONSET, BY REGION, 2018-2030 (USD MILLION)
• TABLE 10. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 11. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY ANTICONVULSANT AGENTS, BY REGION, 2018-2030 (USD MILLION)
• TABLE 12. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY BONE MARROW TRANSPLANTATION, BY REGION, 2018-2030 (USD MILLION)
• TABLE 13. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY MUSCLE RELAXER DRUGS, BY REGION, 2018-2030 (USD MILLION)
• TABLE 14. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 15. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY E-COMMERCE, BY REGION, 2018-2030 (USD MILLION)
• TABLE 16. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY HOSPITAL PHARMACIES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 17. GLOBAL KRABBE DISEASE TREATMENT MARKET SIZE, BY RETAIL PHARMACIES, BY REGION, 2018-2030 (USD MILLION)
• TABLE 18. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 19. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 20. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 21. AMERICAS KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 22. ARGENTINA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 23. ARGENTINA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 24. ARGENTINA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 25. BRAZIL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 26. BRAZIL KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 27. BRAZIL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 28. CANADA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 29. CANADA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 30. CANADA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 31. MEXICO KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 32. MEXICO KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 33. MEXICO KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 34. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 35. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 36. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 37. UNITED STATES KRABBE DISEASE TREATMENT MARKET SIZE, BY STATE, 2018-2030 (USD MILLION)
• TABLE 38. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 39. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 40. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 41. ASIA-PACIFIC KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 42. AUSTRALIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 43. AUSTRALIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 44. AUSTRALIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 45. CHINA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 46. CHINA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 47. CHINA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 48. INDIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 49. INDIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 50. INDIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 51. INDONESIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 52. INDONESIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 53. INDONESIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 54. JAPAN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 55. JAPAN KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 56. JAPAN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 57. MALAYSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 58. MALAYSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 59. MALAYSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 60. PHILIPPINES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 61. PHILIPPINES KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 62. PHILIPPINES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 63. SINGAPORE KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 64. SINGAPORE KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 65. SINGAPORE KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 66. SOUTH KOREA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 67. SOUTH KOREA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 68. SOUTH KOREA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 69. TAIWAN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 70. TAIWAN KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 71. TAIWAN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 72. THAILAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 73. THAILAND KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 74. THAILAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 75. VIETNAM KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 76. VIETNAM KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 77. VIETNAM KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 78. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 79. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 80. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 81. EUROPE, MIDDLE EAST & AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY COUNTRY, 2018-2030 (USD MILLION)
• TABLE 82. DENMARK KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 83. DENMARK KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 84. DENMARK KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 85. EGYPT KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 86. EGYPT KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 87. EGYPT KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 88. FINLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 89. FINLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 90. FINLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 91. FRANCE KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 92. FRANCE KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 93. FRANCE KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 94. GERMANY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 95. GERMANY KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 96. GERMANY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 97. ISRAEL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 98. ISRAEL KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 99. ISRAEL KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 100. ITALY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 101. ITALY KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 102. ITALY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 103. NETHERLANDS KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 104. NETHERLANDS KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 105. NETHERLANDS KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 106. NIGERIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 107. NIGERIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 108. NIGERIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 109. NORWAY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 110. NORWAY KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 111. NORWAY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 112. POLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 113. POLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 114. POLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 115. QATAR KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 116. QATAR KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 117. QATAR KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 118. RUSSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 119. RUSSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 120. RUSSIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 121. SAUDI ARABIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 122. SAUDI ARABIA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 123. SAUDI ARABIA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 124. SOUTH AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 125. SOUTH AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 126. SOUTH AFRICA KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 127. SPAIN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 128. SPAIN KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 129. SPAIN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 130. SWEDEN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 131. SWEDEN KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 132. SWEDEN KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 133. SWITZERLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 134. SWITZERLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 135. SWITZERLAND KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 136. TURKEY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 137. TURKEY KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 138. TURKEY KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 139. UNITED ARAB EMIRATES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 140. UNITED ARAB EMIRATES KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 141. UNITED ARAB EMIRATES KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 142. UNITED KINGDOM KRABBE DISEASE TREATMENT MARKET SIZE, BY DISEASE TYPE, 2018-2030 (USD MILLION)
• TABLE 143. UNITED KINGDOM KRABBE DISEASE TREATMENT MARKET SIZE, BY TREATMENT, 2018-2030 (USD MILLION)
• TABLE 144. UNITED KINGDOM KRABBE DISEASE TREATMENT MARKET SIZE, BY DISTRIBUTION CHANNEL, 2018-2030 (USD MILLION)
• TABLE 145. KRABBE DISEASE TREATMENT MARKET SHARE, BY KEY PLAYER, 2023
• TABLE 146. KRABBE DISEASE TREATMENT MARKET, FPNV POSITIONING MATRIX, 2023

The Krabbe Disease Treatment Market was valued at USD 1.72 billion in 2023, expected to reach USD 1.83 billion in 2024, and is projected to grow at a CAGR of 6.49%, to USD 2.68 billion by 2030.

Krabbe Disease, a rare and often fatal genetic disorder affecting the nervous system, is increasingly becoming a focal point in healthcare research due to its complexity and limited treatment options. The treatment landscape primarily involves the application of hematopoietic stem-cell transplantation (HSCT), enzyme replacement therapies, and gene therapies, yet the market necessity remains high due to the limited efficacy and availability of these treatments. This unmet need, combined with advancements in biotech and pharmaceuticals, has sparked interest in the development of more effective and accessible solutions. Key growth influencers include increasing awareness and diagnosis of the disease, advances in genetic research, and supportive government policies aimed at rare diseases. Latest opportunities lie in the progression of gene-editing technologies like CRISPR and novel drug formulations, providing potential for groundbreaking treatments and improved quality of life for patients. Nevertheless, the market faces significant challenges, including high R&D costs, regulatory complexities associated with rare diseases, and a limited patient pool, which can deter investment. To overcome these limitations, businesses can focus on collaborative research initiatives and partnerships with academic institutions, which can provide access to cutting-edge technology and shared resources. Innovative efforts in personalized medicine, particularly in tailoring treatments based on genetic profiles, represent an exciting area of potential growth and differentiation. The nature of the market remains both challenging and promising, characterized by rapid technological advances alongside logistical and financial hurdles. Successful market entries and leadership would likely require strategic investment in R&D, agile adaptation to emerging regulatory requirements, and a robust understanding of the patient journey to address critical unmet needs expertly. Engaging in continuous market research to monitor technological trends, policy shifts, and competitive movements will be essential for positioning and sustaining growth in the Krabbe Disease treatment market.

Market Dynamics: Unveiling Key Market Insights in the Rapidly Evolving Krabbe Disease Treatment Market

The Krabbe Disease Treatment Market is undergoing transformative changes driven by a dynamic interplay of supply and demand factors. Understanding these evolving market dynamics prepares business organizations to make informed investment decisions, refine strategic decisions, and seize new opportunities. By gaining a comprehensive view of these trends, business organizations can mitigate various risks across political, geographic, technical, social, and economic domains while also gaining a clearer understanding of consumer behavior and its impact on manufacturing costs and purchasing trends.

• Market Drivers
  • Increasing prevalence of Krabbe disease worldwide
  • Rise in gene therapy and stem cell transplantation research
  • Government and private initiatives to raise awareness about inherited disorders
• Market Restraints
  • High cost associated with treatment for Krabbe disease
• Market Opportunities
  • Advancements in the diagnosis and treatment of Krabbe disease
  • Favorable funding landscape for expanding research on krabbe disease
• Market Challenges
  • Ethical issues with testing and treatment for Krabbe disease

Porter's Five Forces: A Strategic Tool for Navigating the Krabbe Disease Treatment Market

Porter's five forces framework is a critical tool for understanding the competitive landscape of the Krabbe Disease Treatment Market. It offers business organizations with a clear methodology for evaluating their competitive positioning and exploring strategic opportunities. This framework helps businesses assess the power dynamics within the market and determine the profitability of new ventures. With these insights, business organizations can leverage their strengths, address weaknesses, and avoid potential challenges, ensuring a more resilient market positioning.

PESTLE Analysis: Navigating External Influences in the Krabbe Disease Treatment Market

External macro-environmental factors play a pivotal role in shaping the performance dynamics of the Krabbe Disease Treatment Market. Political, Economic, Social, Technological, Legal, and Environmental factors analysis provides the necessary information to navigate these influences. By examining PESTLE factors, businesses can better understand potential risks and opportunities. This analysis enables business organizations to anticipate changes in regulations, consumer preferences, and economic trends, ensuring they are prepared to make proactive, forward-thinking decisions.

Market Share Analysis: Understanding the Competitive Landscape in the Krabbe Disease Treatment Market

A detailed market share analysis in the Krabbe Disease Treatment Market provides a comprehensive assessment of vendors' performance. Companies can identify their competitive positioning by comparing key metrics, including revenue, customer base, and growth rates. This analysis highlights market concentration, fragmentation, and trends in consolidation, offering vendors the insights required to make strategic decisions that enhance their position in an increasingly competitive landscape.

FPNV Positioning Matrix: Evaluating Vendors' Performance in the Krabbe Disease Treatment Market

The Forefront, Pathfinder, Niche, Vital (FPNV) Positioning Matrix is a critical tool for evaluating vendors within the Krabbe Disease Treatment Market. This matrix enables business organizations to make well-informed decisions that align with their goals by assessing vendors based on their business strategy and product satisfaction. The four quadrants provide a clear and precise segmentation of vendors, helping users identify the right partners and solutions that best fit their strategic objectives.

Strategy Analysis & Recommendation: Charting a Path to Success in the Krabbe Disease Treatment Market

A strategic analysis of the Krabbe Disease Treatment Market is essential for businesses looking to strengthen their global market presence. By reviewing key resources, capabilities, and performance indicators, business organizations can identify growth opportunities and work toward improvement. This approach helps businesses navigate challenges in the competitive landscape and ensures they are well-positioned to capitalize on newer opportunities and drive long-term success.

Key Company Profiles

The report delves into recent significant developments in the Krabbe Disease Treatment Market, highlighting leading vendors and their innovative profiles. These include Abbott Laboratories, Acorda Therapeutics Inc., Apteeus SAS, Centogene N.V., Forge Biologics, Inc., Gain Therapeutics, Inc., GlaxoSmithKline Plc, Johnson & Johnson, M6P Therapeutics, Neurogene Inc., Novartis AG, Pfizer Inc., Polaryx Therapeutics, Inc, Polpharma, Sanofi S.A., Takeda Pharmaceutical Company Limited, Teva Pharmaceutical Industries Ltd., and UCB Pharmaceuticals.

Market Segmentation & Coverage

This research report categorizes the Krabbe Disease Treatment Market to forecast the revenues and analyze trends in each of the following sub-markets:

• Based on Disease Type, market is studied across Infantile and Late-onset.
• Based on Treatment, market is studied across Anticonvulsant Agents, Bone Marrow Transplantation, and Muscle Relaxer Drugs.
• Based on Distribution Channel, market is studied across E-commerce, Hospital Pharmacies, and Retail Pharmacies.
• Based on Region, market is studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, Thailand, and Vietnam. The Europe, Middle East & Africa is further studied across Denmark, Egypt, Finland, France, Germany, Israel, Italy, Netherlands, Nigeria, Norway, Poland, Qatar, Russia, Saudi Arabia, South Africa, Spain, Sweden, Switzerland, Turkey, United Arab Emirates, and United Kingdom.

The report offers a comprehensive analysis of the market, covering key focus areas:

1. Market Penetration: A detailed review of the current market environment, including extensive data from top industry players, evaluating their market reach and overall influence.

2. Market Development: Identifies growth opportunities in emerging markets and assesses expansion potential in established sectors, providing a strategic roadmap for future growth.

3. Market Diversification: Analyzes recent product launches, untapped geographic regions, major industry advancements, and strategic investments reshaping the market.

4. Competitive Assessment & Intelligence: Provides a thorough analysis of the competitive landscape, examining market share, business strategies, product portfolios, certifications, regulatory approvals, patent trends, and technological advancements of key players.

5. Product Development & Innovation: Highlights cutting-edge technologies, R&D activities, and product innovations expected to drive future market growth.

The report also answers critical questions to aid stakeholders in making informed decisions:

1. What is the current market size, and what is the forecasted growth?

2. Which products, segments, and regions offer the best investment opportunities?

3. What are the key technology trends and regulatory influences shaping the market?

4. How do leading vendors rank in terms of market share and competitive positioning?

5. What revenue sources and strategic opportunities drive vendors' market entry or exit strategies?

Table of Contents

1. Preface

• 1.1. Objectives of the Study
• 1.2. Market Segmentation & Coverage
• 1.3. Years Considered for the Study
• 1.4. Currency & Pricing
• 1.5. Language
• 1.6. Stakeholders

2. Research Methodology

• 2.1. Define: Research Objective
• 2.2. Determine: Research Design
• 2.3. Prepare: Research Instrument
• 2.4. Collect: Data Source
• 2.5. Analyze: Data Interpretation
• 2.6. Formulate: Data Verification
• 2.7. Publish: Research Report
• 2.8. Repeat: Report Update

3. Executive Summary

4. Market Overview

5. Market Insights

• 5.1. Market Dynamics
  • 5.1.1. Drivers
    • 5.1.1.1. Increasing prevalence of Krabbe disease worldwide
    • 5.1.1.2. Rise in gene therapy and stem cell transplantation research
    • 5.1.1.3. Government and private initiatives to raise awareness about inherited disorders
  • 5.1.2. Restraints
    • 5.1.2.1. High cost associated with treatment for Krabbe disease
  • 5.1.3. Opportunities
    • 5.1.3.1. Advancements in the diagnosis and treatment of Krabbe disease
    • 5.1.3.2. Favorable funding landscape for expanding research on krabbe disease
  • 5.1.4. Challenges
    • 5.1.4.1. Ethical issues with testing and treatment for Krabbe disease
• 5.2. Market Segmentation Analysis
• 5.3. Porter's Five Forces Analysis
  • 5.3.1. Threat of New Entrants
  • 5.3.2. Threat of Substitutes
  • 5.3.3. Bargaining Power of Customers
  • 5.3.4. Bargaining Power of Suppliers
  • 5.3.5. Industry Rivalry
• 5.4. PESTLE Analysis
  • 5.4.1. Political
  • 5.4.2. Economic
  • 5.4.3. Social
  • 5.4.4. Technological
  • 5.4.5. Legal
  • 5.4.6. Environmental

6. Krabbe Disease Treatment Market, by Disease Type

• 6.1. Introduction
• 6.2. Infantile
• 6.3. Late-onset

7. Krabbe Disease Treatment Market, by Treatment

• 7.1. Introduction
• 7.2. Anticonvulsant Agents
• 7.3. Bone Marrow Transplantation
• 7.4. Muscle Relaxer Drugs

8. Krabbe Disease Treatment Market, by Distribution Channel

• 8.1. Introduction
• 8.2. E-commerce
• 8.3. Hospital Pharmacies
• 8.4. Retail Pharmacies

9. Americas Krabbe Disease Treatment Market

• 9.1. Introduction
• 9.2. Argentina
• 9.3. Brazil
• 9.4. Canada
• 9.5. Mexico
• 9.6. United States

10. Asia-Pacific Krabbe Disease Treatment Market

• 10.1. Introduction
• 10.2. Australia
• 10.3. China
• 10.4. India
• 10.5. Indonesia
• 10.6. Japan
• 10.7. Malaysia
• 10.8. Philippines
• 10.9. Singapore
• 10.10. South Korea
• 10.11. Taiwan
• 10.12. Thailand
• 10.13. Vietnam

11. Europe, Middle East & Africa Krabbe Disease Treatment Market

• 11.1. Introduction
• 11.2. Denmark
• 11.3. Egypt
• 11.4. Finland
• 11.5. France
• 11.6. Germany
• 11.7. Israel
• 11.8. Italy
• 11.9. Netherlands
• 11.10. Nigeria
• 11.11. Norway
• 11.12. Poland
• 11.13. Qatar
• 11.14. Russia
• 11.15. Saudi Arabia
• 11.16. South Africa
• 11.17. Spain
• 11.18. Sweden
• 11.19. Switzerland
• 11.20. Turkey
• 11.21. United Arab Emirates
• 11.22. United Kingdom

12. Competitive Landscape

• 12.1. Market Share Analysis, 2023
• 12.2. FPNV Positioning Matrix, 2023
• 12.3. Competitive Scenario Analysis
• 12.4. Strategy Analysis & Recommendation

Companies Mentioned

• 1. Abbott Laboratories
• 2. Acorda Therapeutics Inc.
• 3. Apteeus SAS
• 4. Centogene N.V.
• 5. Forge Biologics, Inc.
• 6. Gain Therapeutics, Inc.
• 7. GlaxoSmithKline Plc
• 8. Johnson & Johnson
• 9. M6P Therapeutics
• 10. Neurogene Inc.
• 11. Novartis AG
• 12. Pfizer Inc.
• 13. Polaryx Therapeutics, Inc
• 14. Polpharma
• 15. Sanofi S.A.
• 16. Takeda Pharmaceutical Company Limited
• 17. Teva Pharmaceutical Industries Ltd.
• 18. UCB Pharmaceuticals