Pompe Disease Market by Product (Alglucosidase Alfa, Avalglucosidase Alfa), Patient Type (Infantile Onset, Late Onset), Treatment Regimen, Distribution Channel - Global Forecast 2025-2032
Description
The Pompe Disease Market was valued at USD 1.53 billion in 2024 and is projected to grow to USD 1.67 billion in 2025, with a CAGR of 9.86%, reaching USD 3.24 billion by 2032.
Comprehensive primer on Pompe disease clinical characteristics, diagnostic imperatives, and evolving therapeutic approaches that shape clinical and commercial strategy
Pompe disease, a rare inherited metabolic disorder driven by deficiency of the lysosomal enzyme acid alpha-glucosidase, presents a complex clinical and operational landscape that demands informed, multidisciplinary responses from clinicians, payers, and industry partners. The condition manifests across a spectrum: infantile presentations often include cardiomyopathy and rapid progression, while late-onset forms primarily feature progressive skeletal muscle weakness and respiratory compromise. Early and accurate diagnosis, supported by newborn screening in some jurisdictions and confirmatory biochemical and genetic testing, remains central to improving outcomes.
Over the last two decades, therapeutic advances have shifted Pompe from a disease with uniformly poor prognosis to one where durable, disease-modifying interventions are available. Enzyme replacement therapies have altered the natural history, expanding survival and functional outcomes, yet they also introduce durable care commitments, lifelong infusion regimens, and specialized delivery infrastructure. Stakeholders must therefore balance clinical efficacy with operational feasibility, patient quality of life, and long-term resource planning. This executive summary synthesizes the clinical context, treatment landscape, regulatory and commercial dynamics, and strategic implications to help decision-makers develop pragmatic, patient-centric strategies.
Strategic industry shifts in therapeutics, regulatory pathways, care delivery, and manufacturing that are reshaping the future of Pompe disease management
The landscape for Pompe disease has undergone transformative shifts driven by biology-informed therapy design, regulatory evolution, and changing care delivery models. One of the most consequential shifts is the refinement of enzyme engineering to improve cellular uptake and distribution, which has broadened the clinical dialogue beyond survival to include functional gains, reduced ventilator dependence, and mobility preservation. Regulators have responded with conditional approvals, label refinements, and adaptive pathways that reflect both the rarity of the disease and the unmet clinical need, thereby enabling earlier patient access to promising therapies.
Concurrently, the rise of multidisciplinary care networks and integrated rare disease centers has reshaped management pathways. These centers consolidate diagnostic expertise, infusion capacity, and rehabilitation services, improving coordination and outcomes. Payer models are also adapting, with an increased focus on real-world evidence generation, outcomes-based contracting, and supportive programs that address adherence and long-term monitoring. Supply chain resilience and manufacturing optimization have become strategic priorities as manufacturers scale biologics production and navigate cold-chain logistics. Taken together, these shifts create new opportunities for differentiated product positioning, innovative reimbursement solutions, and collaborative care models that prioritize sustained clinical benefit and patient experience.
Assessment of how evolving United States tariff policies could influence biologics supply chains, operational costs, and continuity of care for Pompe disease therapies
Trade policy and tariff dynamics can exert indirect yet meaningful pressure on the production and distribution economics for biologic therapies used in Pompe disease, and stakeholders should evaluate these forces as part of strategic planning. Tariff adjustments affecting imported raw materials, specialized reagents, single-use bioprocessing equipment, and ancillary medical supplies can increase procurement complexity and operating costs for manufacturers and healthcare providers. In addition, changes in cross-border duties can lengthen lead times, complicate inventory management, and require contingency sourcing to avoid treatment disruptions for patients on life-sustaining infusion regimens.
From a practical standpoint, manufacturers and care providers should prioritize supply chain mapping, identify critical single-source inputs, and develop redundant supplier networks to mitigate exposure to tariff-induced interruptions. Payers and health systems may need to reassess contracting approaches and logistics strategies to preserve access and affordability. In parallel, advocacy and policy engagement remain important levers: documenting patient impact, operational risks, and clinical consequences of supply constraints can shape policymaker responses and carve-outs for essential biologics. Ultimately, preparedness and scenario planning are essential to preserve continuity of care in the face of evolving tariff environments and trade uncertainties.
Integrated segmentation insights revealing how product characteristics, patient cohorts, dosing schedules, and distribution channels collectively shape therapeutic positioning and care delivery
Segment-level dynamics reveal a nuanced interplay between product profiles, patient cohorts, dosing strategies, and distribution pathways that together influence therapeutic adoption and care design. Based on Product, market analyses typically distinguish between alglucosidase alfa and avalglucosidase alfa, with each molecule presenting distinct biochemical properties, manufacturing footprints, and clinical positioning that inform provider choice and patient response. Based on Patient Type, differentiation between Infantile Onset and Late Onset manifestations drives therapeutic priorities: infantile care emphasizes rapid stabilization and cardiopulmonary outcomes, whereas late-onset management focuses on preserving mobility, respiratory function, and long-term quality of life.
Based on Treatment Regimen, clinical programs and service models often cluster around biweekly infusion protocols versus monthly infusion concepts, and these regimen choices have direct implications for infusion center capacity, patient scheduling, and adherence. Based on Distribution Channel, delivery pathways encompass hospital pharmacy, retail pharmacy, and specialty pharmacy options, each presenting distinct operational requirements, reimbursement interactions, and patient support capabilities. Taken together, these segmentation lenses highlight where clinical differentiation, supply chain design, and service model innovation can create competitive advantage and improve patient-centric outcomes.
Comparative regional perspectives on diagnostic capacity, reimbursement variability, and care network maturity that drive differentiated access strategies across global markets
Regional dynamics in Pompe disease management reflect differences in regulatory frameworks, diagnostic infrastructure, reimbursement models, and care network maturity across the globe. In the Americas, diagnostic adoption is influenced by newborn screening expansion, established referral networks, and payer-driven reimbursement constructs that can accelerate access in jurisdictions with structured rare disease pathways. This region also exhibits robust clinical expertise concentrated in specialized centers, which supports complex infusion therapies and multidisciplinary follow-up.
In Europe, Middle East & Africa, heterogeneous regulatory landscapes and variable healthcare financing mechanisms create a patchwork of access. Some markets demonstrate advanced diagnostic programs and integrated care centers, while others face challenges in supply chain logistics and specialist capacity. Stakeholders in this region must navigate diverse reimbursement criteria and design targeted engagement strategies for payers and clinicians. In Asia-Pacific, increasing investment in rare disease awareness, expansion of diagnostic capabilities, and growing biopharma manufacturing capacity are reshaping the landscape. However, access pathways vary widely between high-income and emerging healthcare systems, so manufacturers and health systems must tailor distribution, patient support, and evidence generation strategies to local realities. Across regions, collaboration between clinical opinion leaders, patient advocacy groups, and payers accelerates guideline development and improves alignment on long-term care requirements.
Corporate strategies and competitive differentiators that combine scientific innovation, manufacturing scale, patient services, and evidence generation to sustain long-term access
Competitive dynamics among companies active in Pompe disease center on scientific differentiation, manufacturing scale, regulatory strategy, and comprehensive patient support. Established biologic providers have leveraged decades of biologics manufacturing experience and global distribution networks to secure treatment continuity while investing in process improvements to enhance consistency and reduce supply volatility. Newer entrants have focused on enzyme optimization, improved receptor-mediated uptake, and formulation refinements to address residual disease burden and to extend clinical benefit in difficult-to-treat tissues.
Commercial strategies increasingly emphasize integrated patient services, including infusion coordination, adherence support, and nurse-led programs that reduce patient burden and improve persistence. Partnerships between manufacturers and specialty pharmacies or hospital systems enable tailored distribution models that reconcile cold-chain requirements with patient convenience. From a regulatory perspective, sustained post-approval evidence generation and label expansions remain important corporate priorities, as do thoughtful pricing and access frameworks that reflect clinical value while addressing payers’ long-term budgetary concerns. In aggregate, the competitive landscape rewards companies that combine scientific differentiation with supply reliability, pragmatic access programs, and a clear commitment to long-term real-world evidence generation.
Practical, high-impact recommendations for manufacturers, payers, and providers to strengthen supply resilience, clinical differentiation, and patient-centered care models
Leaders across industry and healthcare delivery should pursue an action-oriented agenda that aligns clinical impact with operational resilience and sustainable access. First, prioritizing robust supply chain risk management-including dual sourcing for critical reagents, inventory buffers for essential biologics, and strategic geographic diversification of manufacturing-will mitigate exposure to trade-related and logistical disruptions. Second, investing in enhanced enzyme design and delivery technologies that demonstrably improve tissue uptake and functional outcomes will create meaningful clinical differentiation and support durable payer value propositions.
Third, fostering collaborative care models that integrate diagnosis, infusion delivery, rehabilitation, and respiratory care within multidisciplinary centers will improve longitudinal outcomes and streamline resource utilization. Fourth, engaging proactively with payers and regulators to define appropriate real-world endpoints, to support outcomes-based contracting where feasible, and to secure managed access arrangements will reduce access friction. Finally, amplifying patient education and support programs, including adherence facilitation and transition-of-care planning, will improve treatment persistence and quality of life. Collectively, these actions can help align innovation incentives with patient-centric outcomes and system sustainability.
Transparent, multidisciplinary research methodology combining literature synthesis, expert interviews, evidence triangulation, and quality controls to ensure robust insights
The research methodology synthesizes multidisciplinary inputs to generate evidence-based insights while maintaining transparency about sources, assumptions, and limitations. The approach begins with a structured review of clinical literature, regulatory filings, and publicly available guidance documents to establish a rigorous clinical and regulatory foundation. This desk research is complemented by primary qualitative interviews with clinical opinion leaders, infusion center directors, specialty pharmacy executives, and payer representatives to capture operational realities, reimbursement considerations, and unmet needs that are not always visible in published sources.
Findings are triangulated across multiple data streams, including treatment guideline analyses, real-world evidence summaries, and manufacturing and logistics assessments, to ensure coherence and to identify convergent trends. Quality controls include systematic cross-validation of interview inputs, explicit documentation of evidence strength, and sensitivity checks against alternative interpretations. The methodology also acknowledges constraints: access to proprietary commercial data is limited, regional heterogeneity complicates universal generalizations, and rapidly evolving therapeutic pipelines may alter competitive dynamics. Where necessary, caveats are noted and recommendations are framed to be robust under plausible near-term shifts.
Concluding synthesis that links scientific progress, operational readiness, and payer engagement to ensure sustainable patient access and improved clinical outcomes
In conclusion, the Pompe disease landscape has matured from isolated clinical interventions to an ecosystem characterized by engineered biologics, multidisciplinary care delivery, and evolving reimbursement constructs that prioritize long-term outcomes. Scientific progress in enzyme design and delivery has materially changed clinical expectations, but sustaining patient access requires concerted attention to supply chain resilience, tailored distribution models, and meaningful engagement with payers to align long-term value with affordability. Moreover, regional variability in diagnostic capacity and healthcare infrastructure underscores the need for locally adapted strategies that reflect differing pathways to care.
Looking ahead, stakeholders who invest in operational readiness, evidence generation, and collaborative care models will be best positioned to translate therapeutic advances into measurable patient benefit. By integrating clinical insight with pragmatic commercial and policy strategies, providers and industry leaders can strengthen continuity of care, optimize resource allocation, and ultimately improve lives for patients across the Pompe disease spectrum.
Please Note: PDF & Excel + Online Access - 1 Year
Comprehensive primer on Pompe disease clinical characteristics, diagnostic imperatives, and evolving therapeutic approaches that shape clinical and commercial strategy
Pompe disease, a rare inherited metabolic disorder driven by deficiency of the lysosomal enzyme acid alpha-glucosidase, presents a complex clinical and operational landscape that demands informed, multidisciplinary responses from clinicians, payers, and industry partners. The condition manifests across a spectrum: infantile presentations often include cardiomyopathy and rapid progression, while late-onset forms primarily feature progressive skeletal muscle weakness and respiratory compromise. Early and accurate diagnosis, supported by newborn screening in some jurisdictions and confirmatory biochemical and genetic testing, remains central to improving outcomes.
Over the last two decades, therapeutic advances have shifted Pompe from a disease with uniformly poor prognosis to one where durable, disease-modifying interventions are available. Enzyme replacement therapies have altered the natural history, expanding survival and functional outcomes, yet they also introduce durable care commitments, lifelong infusion regimens, and specialized delivery infrastructure. Stakeholders must therefore balance clinical efficacy with operational feasibility, patient quality of life, and long-term resource planning. This executive summary synthesizes the clinical context, treatment landscape, regulatory and commercial dynamics, and strategic implications to help decision-makers develop pragmatic, patient-centric strategies.
Strategic industry shifts in therapeutics, regulatory pathways, care delivery, and manufacturing that are reshaping the future of Pompe disease management
The landscape for Pompe disease has undergone transformative shifts driven by biology-informed therapy design, regulatory evolution, and changing care delivery models. One of the most consequential shifts is the refinement of enzyme engineering to improve cellular uptake and distribution, which has broadened the clinical dialogue beyond survival to include functional gains, reduced ventilator dependence, and mobility preservation. Regulators have responded with conditional approvals, label refinements, and adaptive pathways that reflect both the rarity of the disease and the unmet clinical need, thereby enabling earlier patient access to promising therapies.
Concurrently, the rise of multidisciplinary care networks and integrated rare disease centers has reshaped management pathways. These centers consolidate diagnostic expertise, infusion capacity, and rehabilitation services, improving coordination and outcomes. Payer models are also adapting, with an increased focus on real-world evidence generation, outcomes-based contracting, and supportive programs that address adherence and long-term monitoring. Supply chain resilience and manufacturing optimization have become strategic priorities as manufacturers scale biologics production and navigate cold-chain logistics. Taken together, these shifts create new opportunities for differentiated product positioning, innovative reimbursement solutions, and collaborative care models that prioritize sustained clinical benefit and patient experience.
Assessment of how evolving United States tariff policies could influence biologics supply chains, operational costs, and continuity of care for Pompe disease therapies
Trade policy and tariff dynamics can exert indirect yet meaningful pressure on the production and distribution economics for biologic therapies used in Pompe disease, and stakeholders should evaluate these forces as part of strategic planning. Tariff adjustments affecting imported raw materials, specialized reagents, single-use bioprocessing equipment, and ancillary medical supplies can increase procurement complexity and operating costs for manufacturers and healthcare providers. In addition, changes in cross-border duties can lengthen lead times, complicate inventory management, and require contingency sourcing to avoid treatment disruptions for patients on life-sustaining infusion regimens.
From a practical standpoint, manufacturers and care providers should prioritize supply chain mapping, identify critical single-source inputs, and develop redundant supplier networks to mitigate exposure to tariff-induced interruptions. Payers and health systems may need to reassess contracting approaches and logistics strategies to preserve access and affordability. In parallel, advocacy and policy engagement remain important levers: documenting patient impact, operational risks, and clinical consequences of supply constraints can shape policymaker responses and carve-outs for essential biologics. Ultimately, preparedness and scenario planning are essential to preserve continuity of care in the face of evolving tariff environments and trade uncertainties.
Integrated segmentation insights revealing how product characteristics, patient cohorts, dosing schedules, and distribution channels collectively shape therapeutic positioning and care delivery
Segment-level dynamics reveal a nuanced interplay between product profiles, patient cohorts, dosing strategies, and distribution pathways that together influence therapeutic adoption and care design. Based on Product, market analyses typically distinguish between alglucosidase alfa and avalglucosidase alfa, with each molecule presenting distinct biochemical properties, manufacturing footprints, and clinical positioning that inform provider choice and patient response. Based on Patient Type, differentiation between Infantile Onset and Late Onset manifestations drives therapeutic priorities: infantile care emphasizes rapid stabilization and cardiopulmonary outcomes, whereas late-onset management focuses on preserving mobility, respiratory function, and long-term quality of life.
Based on Treatment Regimen, clinical programs and service models often cluster around biweekly infusion protocols versus monthly infusion concepts, and these regimen choices have direct implications for infusion center capacity, patient scheduling, and adherence. Based on Distribution Channel, delivery pathways encompass hospital pharmacy, retail pharmacy, and specialty pharmacy options, each presenting distinct operational requirements, reimbursement interactions, and patient support capabilities. Taken together, these segmentation lenses highlight where clinical differentiation, supply chain design, and service model innovation can create competitive advantage and improve patient-centric outcomes.
Comparative regional perspectives on diagnostic capacity, reimbursement variability, and care network maturity that drive differentiated access strategies across global markets
Regional dynamics in Pompe disease management reflect differences in regulatory frameworks, diagnostic infrastructure, reimbursement models, and care network maturity across the globe. In the Americas, diagnostic adoption is influenced by newborn screening expansion, established referral networks, and payer-driven reimbursement constructs that can accelerate access in jurisdictions with structured rare disease pathways. This region also exhibits robust clinical expertise concentrated in specialized centers, which supports complex infusion therapies and multidisciplinary follow-up.
In Europe, Middle East & Africa, heterogeneous regulatory landscapes and variable healthcare financing mechanisms create a patchwork of access. Some markets demonstrate advanced diagnostic programs and integrated care centers, while others face challenges in supply chain logistics and specialist capacity. Stakeholders in this region must navigate diverse reimbursement criteria and design targeted engagement strategies for payers and clinicians. In Asia-Pacific, increasing investment in rare disease awareness, expansion of diagnostic capabilities, and growing biopharma manufacturing capacity are reshaping the landscape. However, access pathways vary widely between high-income and emerging healthcare systems, so manufacturers and health systems must tailor distribution, patient support, and evidence generation strategies to local realities. Across regions, collaboration between clinical opinion leaders, patient advocacy groups, and payers accelerates guideline development and improves alignment on long-term care requirements.
Corporate strategies and competitive differentiators that combine scientific innovation, manufacturing scale, patient services, and evidence generation to sustain long-term access
Competitive dynamics among companies active in Pompe disease center on scientific differentiation, manufacturing scale, regulatory strategy, and comprehensive patient support. Established biologic providers have leveraged decades of biologics manufacturing experience and global distribution networks to secure treatment continuity while investing in process improvements to enhance consistency and reduce supply volatility. Newer entrants have focused on enzyme optimization, improved receptor-mediated uptake, and formulation refinements to address residual disease burden and to extend clinical benefit in difficult-to-treat tissues.
Commercial strategies increasingly emphasize integrated patient services, including infusion coordination, adherence support, and nurse-led programs that reduce patient burden and improve persistence. Partnerships between manufacturers and specialty pharmacies or hospital systems enable tailored distribution models that reconcile cold-chain requirements with patient convenience. From a regulatory perspective, sustained post-approval evidence generation and label expansions remain important corporate priorities, as do thoughtful pricing and access frameworks that reflect clinical value while addressing payers’ long-term budgetary concerns. In aggregate, the competitive landscape rewards companies that combine scientific differentiation with supply reliability, pragmatic access programs, and a clear commitment to long-term real-world evidence generation.
Practical, high-impact recommendations for manufacturers, payers, and providers to strengthen supply resilience, clinical differentiation, and patient-centered care models
Leaders across industry and healthcare delivery should pursue an action-oriented agenda that aligns clinical impact with operational resilience and sustainable access. First, prioritizing robust supply chain risk management-including dual sourcing for critical reagents, inventory buffers for essential biologics, and strategic geographic diversification of manufacturing-will mitigate exposure to trade-related and logistical disruptions. Second, investing in enhanced enzyme design and delivery technologies that demonstrably improve tissue uptake and functional outcomes will create meaningful clinical differentiation and support durable payer value propositions.
Third, fostering collaborative care models that integrate diagnosis, infusion delivery, rehabilitation, and respiratory care within multidisciplinary centers will improve longitudinal outcomes and streamline resource utilization. Fourth, engaging proactively with payers and regulators to define appropriate real-world endpoints, to support outcomes-based contracting where feasible, and to secure managed access arrangements will reduce access friction. Finally, amplifying patient education and support programs, including adherence facilitation and transition-of-care planning, will improve treatment persistence and quality of life. Collectively, these actions can help align innovation incentives with patient-centric outcomes and system sustainability.
Transparent, multidisciplinary research methodology combining literature synthesis, expert interviews, evidence triangulation, and quality controls to ensure robust insights
The research methodology synthesizes multidisciplinary inputs to generate evidence-based insights while maintaining transparency about sources, assumptions, and limitations. The approach begins with a structured review of clinical literature, regulatory filings, and publicly available guidance documents to establish a rigorous clinical and regulatory foundation. This desk research is complemented by primary qualitative interviews with clinical opinion leaders, infusion center directors, specialty pharmacy executives, and payer representatives to capture operational realities, reimbursement considerations, and unmet needs that are not always visible in published sources.
Findings are triangulated across multiple data streams, including treatment guideline analyses, real-world evidence summaries, and manufacturing and logistics assessments, to ensure coherence and to identify convergent trends. Quality controls include systematic cross-validation of interview inputs, explicit documentation of evidence strength, and sensitivity checks against alternative interpretations. The methodology also acknowledges constraints: access to proprietary commercial data is limited, regional heterogeneity complicates universal generalizations, and rapidly evolving therapeutic pipelines may alter competitive dynamics. Where necessary, caveats are noted and recommendations are framed to be robust under plausible near-term shifts.
Concluding synthesis that links scientific progress, operational readiness, and payer engagement to ensure sustainable patient access and improved clinical outcomes
In conclusion, the Pompe disease landscape has matured from isolated clinical interventions to an ecosystem characterized by engineered biologics, multidisciplinary care delivery, and evolving reimbursement constructs that prioritize long-term outcomes. Scientific progress in enzyme design and delivery has materially changed clinical expectations, but sustaining patient access requires concerted attention to supply chain resilience, tailored distribution models, and meaningful engagement with payers to align long-term value with affordability. Moreover, regional variability in diagnostic capacity and healthcare infrastructure underscores the need for locally adapted strategies that reflect differing pathways to care.
Looking ahead, stakeholders who invest in operational readiness, evidence generation, and collaborative care models will be best positioned to translate therapeutic advances into measurable patient benefit. By integrating clinical insight with pragmatic commercial and policy strategies, providers and industry leaders can strengthen continuity of care, optimize resource allocation, and ultimately improve lives for patients across the Pompe disease spectrum.
Please Note: PDF & Excel + Online Access - 1 Year
Table of Contents
195 Pages
- 1. Preface
- 1.1. Objectives of the Study
- 1.2. Market Segmentation & Coverage
- 1.3. Years Considered for the Study
- 1.4. Currency
- 1.5. Language
- 1.6. Stakeholders
- 2. Research Methodology
- 3. Executive Summary
- 4. Market Overview
- 5. Market Insights
- 5.1. Expansion of next generation gene replacement therapies targeting acid alpha glucosidase expression in skeletal muscle and cardiac tissue
- 5.2. Growing adoption of home-based enzyme replacement infusion services supported by telehealth monitoring and digital adherence tools
- 5.3. Accelerating newborn screening programs to facilitate early Pompe disease diagnosis and treatment initiation in high risk populations
- 5.4. Emergence of chaperone assisted enzyme stabilization approaches to improve intracellular delivery of therapeutic proteins in Pompe patients
- 5.5. Shift toward value based reimbursement models driven by real world evidence of long term outcomes and healthcare resource utilization
- 5.6. Development of investigational CRISPR based gene editing candidates aimed at durable correction of GAA gene mutations for infantile form Pompe disease
- 5.7. Increasing partnerships between biopharma and patient advocacy groups to co design clinical trials and patient centric care pathways
- 6. Cumulative Impact of United States Tariffs 2025
- 7. Cumulative Impact of Artificial Intelligence 2025
- 8. Pompe Disease Market, by Product
- 8.1. Alglucosidase Alfa
- 8.2. Avalglucosidase Alfa
- 9. Pompe Disease Market, by Patient Type
- 9.1. Infantile Onset
- 9.2. Late Onset
- 10. Pompe Disease Market, by Treatment Regimen
- 10.1. Biweekly Infusion
- 10.2. Monthly Infusion
- 11. Pompe Disease Market, by Distribution Channel
- 11.1. Hospital Pharmacy
- 11.2. Retail Pharmacy
- 11.3. Specialty Pharmacy
- 12. Pompe Disease Market, by Region
- 12.1. Americas
- 12.1.1. North America
- 12.1.2. Latin America
- 12.2. Europe, Middle East & Africa
- 12.2.1. Europe
- 12.2.2. Middle East
- 12.2.3. Africa
- 12.3. Asia-Pacific
- 13. Pompe Disease Market, by Group
- 13.1. ASEAN
- 13.2. GCC
- 13.3. European Union
- 13.4. BRICS
- 13.5. G7
- 13.6. NATO
- 14. Pompe Disease Market, by Country
- 14.1. United States
- 14.2. Canada
- 14.3. Mexico
- 14.4. Brazil
- 14.5. United Kingdom
- 14.6. Germany
- 14.7. France
- 14.8. Russia
- 14.9. Italy
- 14.10. Spain
- 14.11. China
- 14.12. India
- 14.13. Japan
- 14.14. Australia
- 14.15. South Korea
- 15. Competitive Landscape
- 15.1. Market Share Analysis, 2024
- 15.2. FPNV Positioning Matrix, 2024
- 15.3. Competitive Analysis
- 15.3.1. Sanofi S.A.
- 15.3.2. Amicus Therapeutics, Inc.
- 15.3.3. Shionogi Inc.
- 15.3.4. Maze Therapeutics
- 15.3.5. GeneCradle Pharmaceutical
- 15.3.6. BioMarin Pharmaceutical Inc.
- 15.3.7. Audentes Therapeutics Inc.
- 15.3.8. AVROBIO, Inc.
- 15.3.9. EpiVax Inc.
- 15.3.10. Valerion Therapeutics LLC
- 15.3.11. Orchard Therapeutics
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