Ornithine Transcarbamylase Deficiency (OTCD) Market to Expand at a Significant Growth Rate During the Forecast Period (2023-32) - Estimates DelveInsight | Key Companies - Arcturus Therapeutics (ARCT-810), Ultragenyx (DTX301)

DelveInsight's "Ornithine Transcarbamylase Deficiency Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Ornithine Transcarbamylase Deficiency market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The report covers emerging Ornithine Transcarbamylase Deficiency drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Ornithine Transcarbamylase Deficiency treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Ornithine Transcarbamylase Deficiency (OTCD): An Overview
According to NORD, Ornithine transcarbamylase (OTC/OTCD) deficiency is a rare X-linked genetic disorder characterized by complete or partial lack of the enzyme ornithine transcarbamylase (OTC) and is the only X-linked urea cycle disorder. The disorder that prevents the breakdown and excretion of ammonia; this allows ammonia to rise to toxic levels and affect the central nervous system.

OTC is one of six enzymes that play a role in the break down and removal of nitrogen from the body, a process known as the urea cycle. The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood. Excess ammonia, which is a neurotoxin, travels to the central nervous system through the blood, resulting in the symptoms and physical findings associated with OTC deficiency.

The underlying cause of OTC deficiency is a gene mutation on the X chromosome and over 100 mutations have been found to result in OTCD. Patients inherit one of these mutations, or a mutation occurs de novo in his/her genome. OTC deficiency occurs more commonly in neonates and early childhood than in adulthood. Females that have a defective gene present on one of their X chromosomes are carriers for that disorder. Carrier females usually do not display symptoms because females have two X chromosomes and only one carries the defective gene.

Treatment for OTC deficiency includes hydration, arginine, and hemodialysis. Long-term therapy for OTC deficiency combines dietary restrictions and the stimulation of alternative methods of converting and excreting nitrogen from the body (alternative pathways therapy).

Ornithine Transcarbamylase Deficiency (OTCD) Market Key Facts
• Stone et al., (2020) in the study titled "Urea Cycle Disorders" stated that about two-thirds of all UCDs are due to mutations in OTC, one-fifth due to argininosuccinate synthase 1 (ASS1), and one-tenth due to argininosuccinate lyase (ASL).
• According to NORD, OTC deficiency affects males more often than females and is fully expressed in males only. In males, symptoms typically begin during the first few days of life. Late-onset OTC deficiency can present later in childhood, but may also occur with onset at 40-50 years of age. Approximately 20% of carrier females have mild symptoms of the disorder and rarely may be severely affected in childhood. The estimated frequency of OTC deficiency is 1/50,000-80,000. The estimated frequency of urea cycle disorders collectively is 1/35,000.

Get More Insights into the Evolving Ornithine Transcarbamylase Deficiency Therapeutics Market Landscape:
https://www.delveinsight.com/report-store/ornithine-transcarbamylase-deficiency-otc-deficiency-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=rpr

Ornithine Transcarbamylase Deficiency (OTCD) Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Ornithine Transcarbamylase Deficiency pipeline therapies. It also thoroughly assesses the Ornithine Transcarbamylase Deficiency market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete detail of the market trend for each marketed Ornithine Transcarbamylase Deficiency drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Ornithine Transcarbamylase Deficiency (OTCD) Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Ornithine Transcarbamylase Deficiency epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Ornithine Transcarbamylase Deficiency epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.

The Report Covers the Ornithine Transcarbamylase Deficiency Epidemiology, Segmented as -
• Prevalent Population of Urea Cycle Disorder (UCD) in the 7MM (2019-2032)
• Diagnosed and Treatable Cases of Ornithine Transcarbamylase Deficiency in the 7MM (2019-2032)
• Prevalent Population of Ornithine Transcarbamylase Deficiency in the 7MM (2019-2032)

Learn more about the Ornithine Transcarbamylase Deficiency (OTCD) Epidemiology Forecast and Key Trends:
https://www.delveinsight.com/report-store/ornithine-transcarbamylase-deficiency-otc-deficiency-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=rpr

Ornithine Transcarbamylase Deficiency (OTCD) Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Ornithine Transcarbamylase Deficiency market or expected to be launched during the study period. The analysis covers the Ornithine Transcarbamylase Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Ornithine Transcarbamylase Deficiency drugs based on their sale and market share.

The report also covers the Ornithine Transcarbamylase Deficiency pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Ornithine Transcarbamylase Deficiency companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Learn How the Ornithine Transcarbamylase Deficiency Market Will Evolve and Grow by 2032:
https://www.delveinsight.com/sample-request/ornithine-transcarbamylase-deficiency-otc-deficiency-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=rpr

Ornithine Transcarbamylase Deficiency (OTCD) Therapeutics Analysis
The pipeline of Ornithine Transcarbamylase Deficiency possess very few potential key players, such as Arcturus Therapeutics, Ultragenyx and others. The dynamics of the OCT deficiency market are anticipated to change in the coming years owing to the improvement in the research and development activities so that the market will be composed of efficient treatment options. The launch of emerging therapies is expected in the near future.

Ornithine Transcarbamylase Deficiency (OTCD) Companies Actively Working in the Therapeutics Market Include
• Arcturus Therapeutics
• Ultragenyx
And others

Emerging and Marketed Ornithine Transcarbamylase Deficiency (OTCD) Therapies Covered in the Report Include:
• ARCT-810: Arcturus Therapeutics
Arcturus Therapeutics is developing ARCT-810, the company's messenger RNA (mRNA)-based therapeutic candidate for OCT deficiency. ARCT-810 utilizes Arcturus' LUNAR (Lipid-enabled and Unlocked Nucleomonomer Agent modified RNA) lipid-mediated delivery platform to effectively deliver OTC messenger RNA to liver cells and is based on Arcturus mRNA design construct and proprietary manufacturing process.

• DTX301: Ultragenyx
DTX301 is an investigational adeno-associated virus 8 (AAV8) gene therapy being developed by Ultragenyx for the treatment of individuals with OTC deficiency. The drug is designed to deliver OTC gene expression in a durable fashion, with the goal of preventing or reducing the occurrence of complications associated with OTC deficiency. Additionally, the drug was granted Orphan Drug Designation in both the US and Europe and the United Kingdom and Fast Track Designation in the US.

The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More:
https://www.delveinsight.com/sample-request/ornithine-transcarbamylase-deficiency-otc-deficiency-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=rpr

Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Ornithine Transcarbamylase Deficiency Competitive Intelligence Analysis
4. Ornithine Transcarbamylase Deficiency Market Overview at a Glance
5. Ornithine Transcarbamylase Deficiency Disease Background and Overview
6. Ornithine Transcarbamylase Deficiency Patient Journey
7. Ornithine Transcarbamylase Deficiency Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Ornithine Transcarbamylase Deficiency Treatment Algorithm, Current Treatment, and Medical Practices
9. Ornithine Transcarbamylase Deficiency Unmet Needs
10. Key Endpoints of Ornithine Transcarbamylase Deficiency Treatment
11. Ornithine Transcarbamylase Deficiency Marketed Therapies
12. Ornithine Transcarbamylase Deficiency Emerging Drugs and Latest Therapeutic Advances
13. Ornithine Transcarbamylase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Ornithine Transcarbamylase Deficiency Market Outlook (In US, EU5, and Japan)
16. Ornithine Transcarbamylase Deficiency Companies Active in the Market
17. Ornithine Transcarbamylase Deficiency Access and Reimbursement Overview
18. KOL Views on the Ornithine Transcarbamylase Deficiency Market
19. Ornithine Transcarbamylase Deficiency Market Drivers
20. Ornithine Transcarbamylase Deficiency Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

Download the Sample PDF to Learn More About the Key Offerings of the Report:
https://www.delveinsight.com/sample-request/ornithine-transcarbamylase-deficiency-otc-deficiency-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=rpr

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.

-------------------------------------------- xx --------------------------------------------
Other Trending Healthcare Reports by DelveInsight
Hairy Cell Leukemia (HCL) Market
https://www.delveinsight.com/report-store/hairy-cell-leukemia-hcl-market

Cancer Cachexia (CC) Market
https://www.delveinsight.com/report-store/cancer-cachexia-cc-market-insights

Diabetes Market
https://www.delveinsight.com/report-store/diabetes-market

Panuveitis Market
https://www.delveinsight.com/report-store/panuveitis-market

Global Kinase Inhibitor in Autoimmune Diseases Market
https://www.delveinsight.com/report-store/global-kinase-inhibitor-in-autoimmune-diseases-market

mRNA Vaccine and Therapeutics Market
https://www.delveinsight.com/report-store/mrna-vaccine-and-therapeutics-market

Negative Pressure Wound Therapy Systems Market
https://www.delveinsight.com/report-store/negative-pressure-wound-therapy-systems-market

Hematuria Market
https://www.delveinsight.com/report-store/hematuria-market

Smart Inhalers Market
https://www.delveinsight.com/report-store/smart-inhalers-market

Audiology Devices Market
https://www.delveinsight.com/report-store/audiology-devices-market

Deep Brain Stimulation Devices Market
https://www.delveinsight.com/report-store/deep-brain-stimulation-devices-market

Prosthetic Heart Valve Market
https://www.delveinsight.com/report-store/prosthetic-heart-valve-market

Tongue Cancer Market
https://www.delveinsight.com/report-store/tongue-cancer-market

Cancer Vaccines Market
https://www.delveinsight.com/report-store/cancer-vaccines-market

CAR-T Pipeline Insight
https://www.delveinsight.com/report-store/car-t-pipeline-insight

Concussions Market
https://www.delveinsight.com/report-store/concussions-market

Total Knee Arthroplasty Market
https://www.delveinsight.com/report-store/total-knee-arthroplasty-market

Physiotherapy Equipment Market
https://www.delveinsight.com/report-store/physiotherapy-equipment-market

Dysthymia Market
https://www.delveinsight.com/report-store/dysthymia-market

Persistent Depressive Disorder Market
https://www.delveinsight.com/report-store/persistent-depressive-disorder-market

Anti-hypertension Market
https://www.delveinsight.com/report-store/anti-hypertension-market

Seborrhoeic Dermatitis Market
https://www.delveinsight.com/report-store/seborrhoeic-dermatitis-market

Spirometers Market
https://www.delveinsight.com/report-store/spirometers-market

Tumor Ablation Market
https://www.delveinsight.com/report-store/tumor-ablation-market

Migraine Market
https://www.delveinsight.com/report-store/migraine-market

Pegfilgrastim Biosimilar
https://www.delveinsight.com/report-store/pegfilgrastim-biosimilar-insight

Renal Vasculitis Market
https://www.delveinsight.com/report-store/renal-vasculitis-market

Dense Deposit Disease Market
https://www.delveinsight.com/report-store/dense-deposit-disease-market

Indolent Lymphoma Market
https://www.delveinsight.com/report-store/indolent-lymphoma-market

Lipodystrophy Market
https://www.delveinsight.com/report-store/lipodystrophy-market

Vutrisiran Market
https://www.delveinsight.com/report-store/vutrisiran-market

Contact Info:
Shruti Thakur
Manager - Marketing & International Branding
Email: info@delveinsight.com

428, D21 Corporate Park, Sector-21, Dwarka, New Delhi-110077, India

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.

This release was published on openPR.