Sickle Cell Disease - Epidemiology Forecast - 2034

Key Highlights

• Due to heightened awareness, resulting in a rise in the diagnosed prevalence of MOCOD-A, analysts anticipate that the Total Prevalent Cases of Molybdenum Cofactor Deficiency Type A (MoCoD-A) in the 7MM stood at approximately 270 in 2023. These numbers are expected to continue increasing throughout the forecast period spanning from 2024 to 2034.
• It is estimated that approximately 54% of the Total Diagnosed Prevalent Cases of Molybdenum Cofactor Deficiency Type A (MoCoD-A) in the 7MM were in the United States. Our estimations indicate that in 2023, the EU4 and the UK collectively represented 36% diagnosed prevalent cases of MOCOD-A.
• According to the analyst's analysis, the Total Diagnosed Prevalent Cases of MOCOD-A in the US was found to be nearly 30 in 2023 and is estimated to increase with a significant CAGR throughout the forecast period.
• As per the analyst's estimates, Japan accounted for nearly 9% of the Total Diagnosed Prevalent Cases of MOCOD-A in the 7MM in 2023.

This report delivers an in-depth understanding of Molybdenum Cofactor Deficiency Type A (MoCoD-A), historical and forecasted epidemiology of Molybdenum Cofactor Deficiency Type A (MoCoD-A) in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Disease Understanding

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Overview

Molybdenum cofactor deficiency (MoCoD) is an uncommon metabolic disorder marked by severe progressive neurological damage, abnormal autonomic function, heightened startle responses, facial abnormalities, changes in muscle tone, advancing cerebral palsy, small head size, seizures, and premature death. It arises from the loss of function of sulfite oxidase, one of four molybdenum-dependent enzymes in humans. MoCoD-A, a subtype of MoCoD, is characterized by persistent seizures, feeding difficulties, and profound neurological impairment, often resulting in early childhood fatality. Due to its rarity, understanding the epidemiology of MoCoD-A poses significant challenges.

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Diagnosis

Diagnosing MoCoD involves identifying biallelic pathogenic variants in GPHN, MOCS1, MOCS2, or MOCS3 genes. Alternatively, if genetic testing is unavailable, significantly reduced sulfite oxidase enzyme activity in cultured fibroblasts confirms the diagnosis. However, distinguishing between total and partial enzyme activity loss is challenging due to low sulfite oxidase expression in fibroblasts. Molecular genetic testing, which is highly sensitive, usually replaces enzymatic testing and is therefore preferred for MoCoD diagnosis.

MoCoD-A diagnosis is complex due to its rarity, vague symptoms, and the requirement for genetic confirmation. Confirming MoCoD-A involves identifying biallelic mutations in the MOCS1 gene and detecting specific metabolites in blood or urine tests. These steps necessitate specialized genetic testing and biochemical analyses.

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Epidemiology

For the purpose of designing the patient-based model for Molybdenum Cofactor Deficiency Type A (MoCoD-A), the report provides historical as well as forecasted epidemiology segmented by Prevalent cases of MoCoD, Diagnosed Prevalent Cases of MoCoD, and Type-specific Diagnosed Prevalent Cases of MoCoD in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan, from 2020 to 2034.

The analyst'sanalyst estimate that approximately 270 Prevalent Cases of MOCOD-A were found in 2023 in the 7MM.

The United States exhibited the highest diagnosed prevalent population of MOCOD-A, as compared to other 7MM countries. As per the analyst's estimations, the Total Diagnosed Prevalent Cases of MOCOD-A in the US was around 30 in 2023 and is projected to increase during the forecast period owing to the increasing awareness among the patient population.

According to This reported in Spain among the EU4 countries.

The data indicates that Japan had the lowest number of Total Diagnosed Prevalent Cases of MOCOD-A among the 7MM in 2023, accounting for just 9% of the overall cases. However, it is projected to experience substantial growth by 2034, with a notable CAGR.

The Diagnosed Prevalence of MOCOD-A was divided into two categories: 'MoCoD Type A' and 'MoCoD Type B and Type C'. MoCoD Type A had a higher number of cases compared to MoCoD Type B and Type C, mainly because mutations in the MOCS1 gene are more prevalent than mutations in the MOCS2 or GPHN genes, which are associated with MoCoD Type B and Type C, respectively.

KOL Views

To gaze into the epidemiology insights of the real world, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research on disease prevalence.

The analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the Children's Hospital Colorado, University of Colorado Denver, United States, University of Cologne, Germany, Department of Pediatrics, Seirei-Mikatahara General Hospital, Japan, and others were contacted. Their opinion helps understand and validate current disease prevalence, gender involved with the disease, diagnosis rate, and diagnostic criteria.

Scope of the Report

• The report covers a segment of key events, an executive summary, descriptive overview of Molybdenum Cofactor Deficiency Type A (MoCoD-A), explaining its causes, signs and symptoms, and currently available diagnostic algorithms and guidelines.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and diagnosis guidelines.
• The report provides an edge for understanding trends, expert insights/KOL views, and patient journeys in the 7MM.
• A detailed review of current challenges in establishing the diagnosis.

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Report Insights

• Patient Population
• Country-wise Epidemiology Distribution
• Prevalent cases of MoCoD
• Diagnosed Prevalent Cases of MoCoD
• Type-specific Diagnosed Prevalent Cases of MoCoD

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Report Key Strengths

• 11 years Forecast
• The 7MM Coverage
• Molybdenum Cofactor Deficiency Type A (MoCoD-A) Epidemiology Segmentation

Molybdenum Cofactor Deficiency Type A (MoCoD-A) Report Assessment

• Current Diagnostic Practices Patient Segmentation

Epidemiology Insights

• What are the disease risk, burdens, and unmet needs of Molybdenum Cofactor Deficiency Type A (MoCoD-A)? What will be the growth opportunities across the 7MM concerning the patient population of Molybdenum Cofactor Deficiency Type A (MoCoD-A)?
• What is the historical and forecasted Molybdenum Cofactor Deficiency Type A (MoCoD-A) patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
• Why is the diagnosed prevalent cases of MOCOD-A in Japan lower than the US?
• Which country has a high patient share for MOCOD-A?

Reasons to Buy

• Insights on patient burden/disease, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the Molybdenum Cofactor Deficiency Type A (MoCoD-A) prevalence cases in varying geographies over the coming years.
• A detailed overview of Type-specific diagnosed prevalence of MOCOD-A.
• To understand the perspective of key opinion leaders around the current challenges with establishing the diagnosis options.
• Detailed insights on various factors hampering disease diagnosis and other existing diagnostic challenges.

Frequently Asked Questions

1. What is the forecast period covered in the report?

The Molybdenum Cofactor Deficiency Type A (MoCoD-A) Epidemiology report for the 7MM covers the forecast period from 2024 to 2034, providing a projection of epidemiology dynamics and trends during this timeframe.

2. Out of all EU4 countries and the UK, which country had the highest population of Molybdenum Cofactor Deficiency Type A (MoCoD-A) cases in 2023?

The highest cases of Molybdenum Cofactor Deficiency Type A (MoCoD-A) was found in the Germany among EU4 and the UK in 2023.

3. How is epidemiological data collected and analyzed for forecasting purposes?

Epidemiological data is collected through surveys, clinical studies, health records, and other sources. It is then analyzed to calculate disease rates, identify trends, and project future disease burdens using mathematical models.

4. Out of all 7MM countries, which country had the highest population of Molybdenum Cofactor Deficiency Type A (MoCoD-A) cases in 2023?

The highest cases of Molybdenum Cofactor Deficiency Type A (MoCoD-A) were found in the US among the 7MM in 2023.