Latest news with #NipponShinyaku
Yahoo
14-05-2025
- Business
- Yahoo
Capricor Therapeutics Inc (CAPR) Q1 2025 Earnings Call Highlights: Navigating Challenges with ...
Cash Position: Approximately $144.8 million as of March 31, 2025. Revenue: $0 for Q1 2025, compared to approximately $4.9 million for Q1 2024. Research and Development Expenses: Approximately $16.2 million for Q1 2025, compared to approximately $10.1 million in Q1 2024. General and Administrative Expenses: Approximately $3.1 million for Q1 2025, compared to approximately $1.8 million in Q1 2024. Net Loss: Approximately $24.4 million for Q1 2025, compared to a net loss of approximately $9.8 million for Q1 2024. Warning! GuruFocus has detected 4 Warning Signs with CAPR. Release Date: May 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Capricor Therapeutics Inc (NASDAQ:CAPR) is on track with its BLA seeking full approval for its product candidate, damy cell, for treating Duchenne muscular dystrophy (DMD) cardiomyopathy. The company has a strong safety record for damy cell, demonstrated in over 700 infusions treating more than 250 patients. Capricor Therapeutics Inc (NASDAQ:CAPR) is transitioning from a translational medicine company to a commercial stage entity, actively working with its commercial partner NS Pharma on launch readiness in the United States. The company has a robust cash position with approximately $145 million, providing a financial runway into 2027 without additional cash infusions. Capricor Therapeutics Inc (NASDAQ:CAPR) is actively expanding its manufacturing capabilities, with plans to operationalize additional clean rooms by mid to late 2026 to meet potential demand. Revenues for the first quarter of 2025 were zero, compared to approximately $4.9 million in the first quarter of 2024. Operating expenses have increased, with research and development expenses rising to approximately $16.2 million in Q1 2025 from $10.1 million in Q1 2024. The net loss for the first quarter of 2025 was approximately $24.4 million, compared to a net loss of $9.8 million in the first quarter of 2024. There is uncertainty regarding the outcome of the FDA advisory committee meeting, which could impact the approval process for damy cell. Negotiations for European distribution with Nippon Shinyaku have been extended, indicating potential delays or challenges in finalizing agreements for the European market. Q: Have you had your site inspection in San Diego, and what are the key features of your preparation for the FDA advisory committee meeting? A: We haven't had our pre-licensing inspection yet, but it's scheduled for this quarter. Our facility was built for commercial manufacturing, so we feel prepared. Regarding the advisory committee meeting, we've had two mock sessions and passed with flying colors. The FDA is actively reviewing our file, and we are confident in our data and preparation. Q: Can you provide an update on the negotiations with Nippon Shinyaku for the European market, and how is NS Pharma preparing for the U.S. launch? A: We are in active negotiations with Nippon Shinyaku for Europe and are also exploring launching on our own. In the U.S., NS Pharma has built a strong sales and marketing team for Viltepso, and they are preparing to launch Daryel with 125 FTEs focusing on it. We are enhancing our management team to support this effort. Q: What are the key drivers that physicians see as proof of the efficacy of your therapy, and will you have four-year data to share at the advisory committee meeting? A: The key driver is the statistical significance of cardiac MRI data, which objectively measures cardiac function. We plan to present four-year data at the PPMG meeting in June, showing long-term stabilization in cardiac function, which is promising. Q: If the FDA issues a CRL for efficacy, what is your plan? Would you submit HOPE 3 data for DMD skeletal muscle function? A: Yes, if a CRL is issued, we would submit the HOPE 3 data for skeletal muscle dysfunction, as it is fully enrolled and expected to be positive. We would apply for both cardiac and skeletal indications based on the data. Q: How does the FDA view LVF as a surrogate endpoint, and how are you supporting this with your data? A: The FDA does not view ejection fraction as a surrogate endpoint in this rare disease context. Instead, they consider it an outcome measure. We have used real-world evidence and natural history data to support our efficacy claims, showing significant improvement in treated patients. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
13-05-2025
- Business
- Yahoo
Regenxbio Inc (RGNX) Q1 2025 Earnings Call Highlights: Strategic Partnerships and Financial ...
Cash, Cash Equivalents, and Marketable Securities: $272 million as of March 31, 2025, compared to $245 million as of December 31, 2024. Upfront Payment: $110 million received under the Nippon Shinyaku collaboration. R&D Expenses: $53 million for the quarter ended March 31, 2025, compared to $54.8 million for the quarter ended March 31, 2024. Cash Runway Guidance: Expected to fund operations into the second half of 2026, excluding potential future royalty income. Warning! GuruFocus has detected 4 Warning Signs with RGNX. Release Date: May 12, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Regenxbio Inc (NASDAQ:RGNX) has made significant progress in its late-stage clinical programs, particularly with RGX-121 for MPS II, which is on track for potential FDA approval in the second half of 2025. The company has established a strategic partnership with Nippon Shinyaku to commercialize its neurodegenerative franchise, enhancing its commercial readiness. RGX-202, a next-generation candidate for Duchenne muscular dystrophy, has surpassed 50% enrollment in its pivotal study, with strong interest from the patient community. Regenxbio Inc (NASDAQ:RGNX) has a state-of-the-art manufacturing facility capable of producing up to 2,500 doses of RGX-202 annually, ensuring readiness for commercial launch. The company's financial position is strong, with $272 million in cash and marketable securities, bolstered by a $110 million upfront payment from Nippon Shinyaku, providing a cash runway into the second half of 2026. The acceptance of the BLA for RGX-121 by the FDA is still pending, creating uncertainty around the timing of regulatory approval. There are concerns about potential changes in FDA requirements for safety profiles, especially in light of recent safety events in the gene therapy space. The company faces competition in the Duchenne muscular dystrophy market, with questions about the approvability of RGX-202 on an accelerated basis. Regenxbio Inc (NASDAQ:RGNX) is navigating a complex regulatory environment with multiple interactions with the FDA, which could impact timelines. The potential impact of recent announcements on drug pricing could affect the company's ability to price its gene therapies competitively. Q: Can you provide an update on the timing for the Hunter BLA acceptance? A: Curran Simpson, President and CEO, stated that the BLA acceptance is imminent. The review process is proceeding normally, with regular interactions and information requests from the FDA. They expect to announce acceptance soon. Q: How do you view the bar for approvability in Duchenne muscular dystrophy (DMD) on an accelerated basis, especially in light of recent industry developments? A: Curran Simpson explained that they have always aimed for an accelerated approval pathway, with discussions with the FDA being positive. They are confident in their data, including safety and initial functional data, and expect to provide further updates in the first half of the year. Q: Are there any expected changes from the FDA regarding safety profile requirements for your pivotal study, especially after recent safety events in the industry? A: Curran Simpson noted that their program is on track, with no anticipated changes in safety requirements. They are confident in their immune suppression regimen and safety profile, which have been well-received by investigators and the patient community. Q: Can you provide more details on the planning process with AbbVie for the diabetic retinopathy Phase 3 trial? A: Curran Simpson mentioned that they are finalizing feedback from regulatory agencies and expect to begin site activation this year. They are considering established endpoints for the pivotal protocol and will provide more specific timing as they approach the first patient dose. Q: What are the expectations for the RGX-202 commercial supply manufacturing process? A: Curran Simpson confirmed that the commercial production process will be the same as the current clinical trials. They plan to initiate commercial production this year, with the process being validated for the mid-2026 BLA submission. The manufacturing process is mature and produces high-purity capsids. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Malaysian Reserve
07-05-2025
- Business
- Malaysian Reserve
Mucopolysaccharidosis Type I Market to Witness Upsurge in Growth at a CAGR of 8% During the Forecast Period (2025-2034)
The future of MPS I treatment is poised for significant advancements, driven by ongoing research and innovative therapies. Emerging therapies, such as stem cell gene therapy, IDUA Gene therapy, and Recombinant DNA, including OTL-203, Lepunafusp alfa (JR-171), RGX-111, and Iduronicrin genleukocel-T (ISP-001), offer novel approaches. These therapies have the potential to reshape the treatment landscape by improving patient outcomes and reducing the limitations of existing treatments. LAS VEGAS, May 7, 2025 /PRNewswire/ — DelveInsight's Mucopolysaccharidosis Type I Market Insights report includes a comprehensive understanding of current treatment practices, Mucopolysaccharidosis type I emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Mucopolysaccharidosis Type I Market Report According to DelveInsight's analysis, the market size for Mucopolysaccharidosis type I was found to be USD 145 million in the 7MM in 2024. in the 7MM in 2024. In 2024, the MPS I market size in the US was highest, accounting for 48% of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies. of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies. According to DelveInsight's estimates, in 2024, there were approximately 660 diagnosed prevalent cases of MPS I in the 7MM. Of these, the United States accounted for 36% of the cases. diagnosed prevalent cases of MPS I in the 7MM. Of these, accounted for of the cases. Leading Mucopolysaccharidosis type I companies developing emerging therapies, such as Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years. and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years. The promising Mucopolysaccharidosis type I therapies in the pipeline include OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others. and others. In March 2025 , REGENXBIO Inc. finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in the United States and Asia . finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in and . In February 2025 , at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study. at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study. In January 2025 , Immusoft announced positive results from the first engineered B Cell (ISP-001) in a human clinical trial, to be presented at the WORLDSymposium. Discover which therapies are expected to grab the major MPS I market share @ Mucopolysaccharidosis Type I Market Report Mucopolysaccharidosis Type I Overview MPS I is a rare inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), which is essential for the breakdown of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate. When these substances accumulate, they lead to progressive damage in cells and tissues, impacting various organ systems, including the skeletal, cardiovascular, respiratory, and nervous systems. The condition presents as a spectrum, with the most severe form being Hurler syndrome and the mildest form known as Scheie syndrome. Diagnosis of MPS I involves testing for elevated GAG levels, enzyme activity assays, and genetic analysis, all of which support early intervention, prognosis determination, and genetic counseling. Early detection is further aided by newborn screening programs. Ongoing disease management relies on comprehensive assessments such as pulmonary function tests, sleep studies, hearing evaluations, eye exams, imaging of the bones, and cognitive testing. Molecular diagnostics and early screening are critical to improving patient outcomes and guiding long-term care. Mucopolysaccharidosis Type I Epidemiology Segmentation The Mucopolysaccharidosis type I epidemiology section provides insights into the historical and current Mucopolysaccharidosis type I patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Mucopolysaccharidosis type I market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalence of MPS I Severity-specific Diagnosed Prevalent Cases of MPS I Treated Cases of MPS I Hurler Syndrome Treatment Market The treatment landscape for MPS I (Hurler syndrome) is progressing gradually, with growing insights into both the disease's biology and its management. Traditional therapies like ERT and HSCT remain central, but advances in precision medicine and new pharmacological options are beginning to show promise. Despite these developments, a universal, comprehensive treatment approach remains elusive due to the disease's complexity and variability among patients, requiring a tailored, multidisciplinary strategy. Supportive care plays a critical role and includes various surgical procedures such as adenotonsillectomy, hernia repair, ventriculoperitoneal shunting, heart valve replacement, carpal tunnel release, and spinal decompression. Therapies like physical, occupational, and speech therapy, along with respiratory aids like CPAP, hearing devices, and medications for managing pain and gastrointestinal symptoms, are also essential. Vision issues may require corneal transplants, although surgical interventions in Hurler syndrome can be risky due to anesthetic complications. ALDURAZYME (laronidase), a recombinant version of the human IDUA enzyme produced using Chinese hamster ovary cells, was approved in the U.S., EU4, the UK (in 2003), and Japan (in 2006). It is indicated for patients with Hurler and Hurler-Scheie types of MPS I, and for symptomatic Scheie patients with moderate to severe manifestations. While ALDURAZYME carries a boxed warning due to potential safety concerns, it remains the only approved ERT for MPS I, with no biosimilars available despite the expiration of its patent. To know more about ALDURAZYME mechanism of action and laronidase cost, visit @ ALDURAZYME Patent Expiration Mucopolysaccharidosis Type I Pipeline Therapies and Key Companies OTL-203: Orchard Therapeutics/Kyowa Kirin Lepunafusp alfa (JR-171): JCR Pharmaceuticals RGX-111: REGENXBIO/Nippon Shinyaku Iduronicrin genleukocel-T (ISP-001): IMMUSOFT Discover more about MPS I drugs in development @ Mucopolysaccharidosis I Clinical Trials Market Mucopolysaccharidosis Type I Market Dynamics The MPS I market dynamics are expected to change in the coming years. Current treatments for MPS I, including ERT and hematopoietic stem cell transplantation, have demonstrated effectiveness in managing symptoms and improving the quality of life for patients with severe forms of the disease. Advances in genetic testing and the expansion of newborn screening programs have enabled earlier diagnosis, allowing for timely intervention and significantly improved patient outcomes. Increased awareness of MPS I, coupled with enhanced genetic counseling, has further contributed to earlier detection and better disease management. Additionally, the growing interest in rare diseases has attracted increased funding and support for research, accelerating the development of new and potentially more effective treatments for MPS I. These combined efforts are shaping a more promising future for patients and families affected by this rare lysosomal storage disorder. Furthermore, potential therapies are being investigated for the treatment of MPS I, and it is safe to predict that the treatment space will significantly impact the mucopolysaccharidosis therapeutics market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the MPS I market in the 7MM. However, several factors may impede the growth of the MPS I market. ERTs for MPS I, such as intravenous iduronidase, are lifelong treatments typically administered every week. While ERT has shown effectiveness in alleviating many somatic symptoms, it is unable to cross the blood-brain barrier, rendering it ineffective in addressing the neurological manifestations associated with severe forms of MPS I. This represents a significant limitation in the current therapeutic approach. As an autosomal recessive disorder, MPS I requires both parents to be carriers, which complicates family planning and increases the likelihood of recurrence in future pregnancies. Despite the benefits of available treatments like ALDURAZYME, the financial burden associated with long-term therapy remains a challenge for many patients and families. Additionally, serious adverse reactions—including life-threatening anaphylaxis—have been reported in some patients during or up to three hours after ALDURAZYME infusion, underscoring the need for careful monitoring and consideration of risk-benefit balance in treatment planning. Moreover, MPS I treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the MPS I market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the MPS I market growth. Mucopolysaccharidosis Type I Market Report Metrics Details Study Period 2020–2034 Coverage 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Mucopolysaccharidosis Type I Market CAGR 8 % Mucopolysaccharidosis Type I Market Size in 2024 USD 145 Million Key Mucopolysaccharidosis Type I Companies Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, and others Key Pipeline Mucopolysaccharidosis Type I Therapies OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others Scope of the Mucopolysaccharidosis Type I Market Report Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and emerging therapies Mucopolysaccharidosis Type I current marketed and emerging therapies Mucopolysaccharidosis Type I Market Dynamics: Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis Type I Market Access and Reimbursement Download the report to understand which factors are driving MPS I market trends @ Mucopolysaccharidosis Type I Market Trends Table of Contents 1 Key Insights 2 Report Introduction 3 MPS I Market Overview at a Glance 3.1 Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2020 3.2 Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2034 4 Executive Summary 5 Key Events 6 Disease Background and Overview 6.1 Introduction 6.2 Causes and Risk Factors 6.3 Clinical Types 6.4 Symptoms 6.5 Pathogenesis 6.6 Diagnosis 6.6.1 Laboratory Diagnosis 6.6.2 Biomarkers 6.6.3 Diagnostic Algorithm 6.6.4 Diagnostic Guidelines 6.7 Treatment 6.7.1 Treatment Algorithm 6.7.2 Treatment Guidelines 7 Epidemiology and Market Methodology 8 Epidemiology and Patient Population 8.1 Key Findings 8.2 Assumptions and Rationale: 7MM 8.2.1 Diagnosed Prevalent Cases of MPS I 8.2.2 Severity-specific Diagnosed Prevalent Cases of MPS I 8.2.3 Treated Cases of MPS I 8.3 Total Diagnosed Prevalent Cases of MPS I in the 7MM 8.4 The United States 8.4.1 Diagnosed Prevalent Cases of MPS I in the US 8.4.2 Severity-specific Diagnosed Prevalent Cases of MPS I in the US 8.4.3 Treated Cases of MPS I in the US 8.5 EU4 and the UK 8.5.1 Diagnosed Prevalent Cases of MPS I in EU4 and the UK 8.5.2 Severity-specific Diagnosed Prevalent Cases of MPS I in EU4 and the UK 8.5.3 Treated Cases of MPS I in EU4 and the UK 8.6 Japan 8.6.1 Diagnosed Prevalent Cases of MPS I in Japan 8.6.2 Severity-specific Diagnosed Prevalent Cases of MPS I in Japan 8.6.3 Treated Cases of MPS I in Japan 9 Patient Journey 10 Marketed Therapies 10.1 ALDURAZYME (laronidase): BioMarin Pharmaceutical/Sanofi 10.1.1 Product Description 10.1.2 Regulatory Milestones 10.1.3 Other Developmental Activities 10.1.4 Clinical Trials Information 10.1.5 Safety and Efficacy To be continued in the report… 11 Emerging Drug Profiles 11.1 Key Cross Competition of Emerging Drugs 11.2 OTL-203: Orchard Therapeutics/Kyowa Kirin 11.2.1 Drug Description 11.2.2 Other Developmental Activities 11.2.3 Clinical Trials Information 11.2.4 Safety and Efficacy 11.2.5 Analysts' Views 11.3 Lepunafusp alfa (JR-171): JCR Pharmaceuticals 11.3.1 Drug Description 11.3.2 Other Developmental Activities 11.3.3 Clinical Trials Information 11.3.4 Safety and Efficacy 11.3.5 Analysts' Views 11.4 RGX-111: REGENXBIO/Nippon Shinyaku 11.4.1 Drug Description 11.4.2 Other Developmental Activities 11.4.3 Clinical Trials Information 11.4.4 Safety and Efficacy 11.4.5 Analysts' Views 11.5 Iduronicrin genleukocel-T (ISP-001): IMMUSOFT 11.5.1 Drug Description 11.5.2 Other Developmental Activities 11.5.3 Clinical Trials Information 11.5.4 Safety and Efficacy 11.5.5 Analysts' Views To be continued in the report… 12 MPS I: Market Analysis 12.1 Key Findings 12.2 Market Outlook 12.3 Attribute Analysis 12.4 Key Market Forecast Assumptions 12.4.1 Cost Assumptions and Rebates 12.4.2 Pricing Trends 12.4.3 Analogue Assessment 12.4.4 Launch Year and Therapy Uptake 12.5 Total Market Size of MPS I in the 7MM 12.6 Market Size of MPS I by Therapies in the 7MM 12.7 Market Size of MPS I in the United States 12.7.1 Total Market Size of MPS I 12.7.2 Market Size of MPS I by Therapies in the United States 12.8 Market Size of MPS I in EU4 and the UK 12.8.1 Total Market Size of MPS I 12.8.2 Market Size of MPS I by Therapies in EU4 and the UK 12.9 Market Size of MPS I in Japan 12.9.1 Total Market Size of MPS I 12.9.2 Market Size of MPS I by Therapies in Japan 13 Key Opinion Leaders' Views 14 Unmet Needs 15 SWOT Analysis 16 Market Access and Reimbursement 16.1 The United States 16.1.1 CMS 16.2 In EU4 and the UK 16.2.1 Germany 16.2.2 France 16.2.3 Italy 16.2.4 Spain 16.2.5 The United Kingdom 16.3 Japan 16.3.1 MHLW 17 Appendix 17.1 Acronyms and Abbreviations 17.2 Bibliography 17.3 Report Methodology 18 DelveInsight Capabilities 19 Disclaimer 20 About DelveInsight Related Reports Mucopolysaccharidosis Type I Pipeline Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key MPS I companies, including Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, among others. Mucopolysaccharidosis Market Mucopolysaccharidosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS companies, including Takeda Pharmaceutical, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Sanofi, among others. Mucopolysaccharidosis Type II Market Mucopolysaccharidosis Type II Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS II companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, among others. Mucopolysaccharidosis Type III Market Mucopolysaccharidosis Type III Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS III companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Lysogene, Inventiva, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ +14699457679 Logo: View original content:
