Table of Content

1. Key Insights

2. Phosphoglucomutase 1 (PGM1) Deficiency Market Overview at a Glance
2.1. Market Share (%) Distribution of PGM1 Deficiency in 2017
2.2. Market Share (%) Distribution of PGM1 Deficiency in 2028

3. Disease Background and Overview
3.1. Introduction
3.2. Facts about Phosphoglucomutase-1
3.3. Pathophysiology
3.4. Causes
3.5. Symptoms
3.6. Multisystem phenotype in PGM1 deficiency
3.7. Diagnosis

4. Epidemiology and Patient Population
4.1. Key Findings
4.2. KOL’s Views: Epidemiology
4.3. Assumptions and Rationale 7MM

5. 7MM Total Prevalent Patient Population of PGM1 Deficiency

6. Country Wise-Epidemiology of PGM1 Deficiency
6.1. The United States
6.1.1. Total Prevalent Cases of PGM1 Deficiency in the United States
6.1.2. Prevalence of PGM1 Deficiency based on Clinical Manifestations in the United States
6.1.3. Prevalence of PGM1 Deficiency based on Congenital malformations in the United States
6.1.4. Prevalence of PGM1 Deficiency based on Severity in the United States
6.2. EU5
6.2.1. Germany
6.2.2. Total Prevalent Cases of PGM1 Deficiency in Germany
6.2.3. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Germany
6.2.4. Prevalence of PGM1 Deficiency based on Congenital malformations in Germany
6.2.5. Prevalence of PGM1 Deficiency based on Severity in Germany
6.2.6. France
6.2.7. Total Prevalent Cases of PGM1 Deficiency in France
6.2.8. Prevalence of PGM1 Deficiency based on Clinical Manifestations in France
6.2.9. Prevalence of PGM1 Deficiency based on Congenital malformations in France
6.2.10. Prevalence of PGM1 Deficiency based on Severity in France
6.2.11. Italy
6.2.12. Total Prevalent Cases of PGM1 Deficiency in Italy
6.2.13. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Italy
6.2.14. Prevalence of PGM1 Deficiency based on Congenital malformations in Italy
6.2.15. Prevalence of PGM1 Deficiency based on Severity in Italy
6.2.16. Spain
6.2.17. Total Prevalent Cases of PGM1 Deficiency in Spain
6.2.18. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Spain
6.2.19. Prevalence of PGM1 Deficiency based on Congenital malformations in Spain
6.2.20. Prevalence of PGM1 Deficiency based on Severity in Spain
6.2.21. The United Kingdom
6.2.22. Total Prevalent Cases of PGM1 Deficiency in the UK
6.2.23. Prevalence of PGM1 Deficiency based on Clinical Manifestations in The UK
6.2.24. Prevalence of PGM1 Deficiency based on Congenital malformations in The UK
6.2.25. Prevalence of PGM1 Deficiency based on Severity in The UK
6.3. Japan
6.3.1. Total Prevalent Cases of PGM1 Deficiency in Japan
6.3.2. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Japan
6.3.3. Prevalence of PGM1 Deficiency based on Congenital malformations in Japan
6.3.4. Prevalence of PGM1 Deficiency based on Severity in Japan

7. Treatment and Management of PGM1 Deficiency

8. Unmet Needs

9. Emerging Therapies
9.1. CERC-801: Cerecor Inc
9.1.1. Drug Description
9.1.2. Clinical Development
9.1.3. Other Developmental Activities
9.1.4. Product Profile
9.2. ORL-1G - D-galactose: Orpha Labs
9.2.1. Drug Description
9.2.2. Clinical Development
9.2.3. Clinical Trials Information
9.2.4. Product Profile

10. PGM1 Deficiency: 7 Major Market Analysis
10.1. Key Findings
10.2. KOL’s Views: Treatment & Diagnosis
10.3. Market Outlook 7MM
10.4. Market Size of PGM1 Deficiency in 7MM

11. Market Outlook by Country
11.1. United States Market Size
11.1.1. Total Market Size of PGM1 Deficiency
11.1.2. Market Size of PGM1 Deficiency by Therapies
11.2. EU-5 Countries: Market Outlook
11.2.1. Germany
11.2.1.1. Total Market size of PGM1 Deficiency
11.2.1.2. Market Size of PGM1 Deficiency by Therapies
11.2.2. France
11.2.2.1. Total Market Size of PGM1 Deficiency
11.2.2.2. Market Size of PGM1 Deficiency by Therapies
11.2.3. Italy
11.2.3.1. Total Market Size of PGM1 Deficiency
11.2.3.2. Market Size of PGM1 Deficiency by Therapies
11.2.4. Spain
11.2.4.1. Total Market Size of PGM1 Deficiency
11.2.4.2. Market Size of PGM1 Deficiency by Therapies
11.2.5. United Kingdom
11.2.5.1. Total Market Size of PGM1 Deficiency
11.2.5.2. Market Size of PGM1 Deficiency by Therapies
11.3. Japan: Market Outlook
11.3.1. Total Market Size of PGM1 Deficiency
11.3.2. Market Size of PGM1 Deficiency by Therapies

12. Market Drivers

13. Market Barriers

14. Appendix

15. Report Methodology

16. DelveInsight Capabilities

17. Disclaimer

18. About DelveInsight



List of Figures


FIGURE 1: SCHEMATIC REPRESENTATION OF PHOSPHOGLUCOMUTASE 1 (PGM1) KEY ROLE IN METABOLISM AND PROTEIN INTERACTION
FIGURE 2: ROLE OF PHOSPHOGLUCOMUTASE 1 IN SUGAR METABOLISM.
FIGURE 3: (A) N-LINKED GLYCOSYLATION REQUIRES THE NUCLEOTIDE SUGARS CMP SIALIC ACID FIGURE 4: N-LINKED GLYCOSYLATION REQUIRES THE NUCLEOTIDE SUGARS CMP SIALIC ACID
FIGURE 5: ASSOCIATED SIGNS AND SYMPTOMS OF PGM-1 DEFICIENCY
FIGURE 6: PREVALENT PATIENT POPULATION OF PGM1 DEFICIENCY IN 7MM (2017-2028)
FIGURE 7: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE US (2017-2028)
FIGURE 8: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE US (2017-2028)
FIGURE 9: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE US (2017-2028)
FIGURE 10: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE US (2017-2028)
FIGURE 11: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN GERMANY (2017-2028)
FIGURE 12: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN GERMANY (2017-2028)
FIGURE 13: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN GERMANY (2017-2028)
FIGURE 14: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN GERMANY (2017-2028)
FIGURE 15: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN FRANCE (2017-2028)
FIGURE 16: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN FRANCE (2017-2028)
FIGURE 17: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN FRANCE (2017-2028)
FIGURE 18: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN FRANCE (2017-2028)
FIGURE 19: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN ITALY (2017-2028)
FIGURE 20: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN ITALY (2017-2028)
FIGURE 21: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN ITALY (2017-2028)
FIGURE 22: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN ITALY (2017-2028)
FIGURE 23: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN SPAIN (2017-2028)
FIGURE 24: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN SPAIN (2017-2028)
FIGURE 25: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN SPAIN (2017-2028)
FIGURE 26: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN SPAIN (2017-2028)
FIGURE 27: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE UK (2017-2028)
FIGURE 28: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE UK (2017-2028)
FIGURE 29: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE UK (2017-2028)
FIGURE 30: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE UK (2017-2028)
FIGURE 31: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN JAPAN (2017-2028)
FIGURE 32: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN JAPAN (2017-2028)
FIGURE 33: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN JAPAN (2017-2028)
FIGURE 34: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN JAPAN (2017-2028)
FIGURE 35: UNMET NEEDS OF PGM1 DEFICIENCY
FIGURE 36: 7 MAJOR MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
FIGURE 37: THE UNITED STATES MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
FIGURE 38: THE US MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 39: MARKET SIZE OF PGM1 DEFICIENCY IN GERMANY, USD MILLION (2017-2028)
FIGURE 40: GERMANY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 41: MARKET SIZE OF PGM1 DEFICIENCY IN FRANCE USD MILLION (2017-2028)
FIGURE 42: FRANCE MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 43: MARKET SIZE OF PGM1 DEFICIENCY IN ITALY, USD MILLION (2017-2028)
FIGURE 44: ITALY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 45: MARKET SIZE OF PGM1 DEFICIENCY IN SPAIN, USD MILLION (2017-2028)
FIGURE 46: SPAIN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 47: MARKET SIZE OF PGM1 DEFICIENCY IN THE UNITED KINGDOM, USD MILLION (2017-2028)
FIGURE 48: THE UK MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 49: MARKET SIZE OF PGM1 DEFICIENCY IN JAPAN, USD MILLION (2017-2028)
FIGURE 50: JAPAN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 51: MARKET DRIVERS OF PGM1 DEFICIENCY
FIGURE 52: MARKET BARRIERS OF PGM1 DEFICIENCY

 

List of Tables


TABLE 1: PREVALENT PATIENT POPULATION OF PGM1 DEFICIENCY IN 7MM (2017-2028)
TABLE 2: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE US (2017-2028)
TABLE 3: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE US (2017-2028)
TABLE 4: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE US (2017-2028)
TABLE 5: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE US (2017-2028)
TABLE 6: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN GERMANY (2017-2028)
TABLE 7: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN GERMANY (2017-2028)
TABLE 8: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN GERMANY (2017-2028)
TABLE 9: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN GERMANY (2017-2028)
TABLE 10: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN FRANCE (2017-2028)
TABLE 11: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN FRANCE (2017-2028)
TABLE 12: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN FRANCE (2017-2028)
TABLE 13: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN FRANCE (2017-2028)
TABLE 14: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN ITALY (2017-2028)
TABLE 15: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN ITALY (2017-2028)
TABLE 16: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN ITALY (2017-2028)
TABLE 17: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN ITALY (2017-2028)
TABLE 18: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN SPAIN (2017-2028)
TABLE 19: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN SPAIN (2017-2028)
TABLE 20: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN SPAIN (2017-2028)
TABLE 21: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN SPAIN (2017-2028)
TABLE 22: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE UK (2017-2028)
TABLE 23: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE UK (2017-2028)
TABLE 24: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE UK (2017-2028)
TABLE 25: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE UK (2017-2028)
TABLE 26: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN JAPAN (2017-2028)
TABLE 27: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN JAPAN (2017-2028)
TABLE 28: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN JAPAN (2017-2028)
TABLE 29: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN JAPAN (2017-2028)
TABLE 30: ORL-1G - D-GALACTOSE CLINICAL TRIAL DESCRIPTION, 2019
TABLE 31: 7 MAJOR MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 32: THE UNITED STATES MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 33: THE US MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 34: MARKET SIZE OF PGM1 DEFICIENCY IN GERMANY, USD MILLION (2017-2028)
TABLE 35: GERMANY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 36: FRANCE MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 37: FRANCE MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 38: ITALY MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 39: ITALY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 40: SPAIN MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 41: SPAIN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 42: UNITED KINGDOM MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 43: THE UK MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 44: JAPAN MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 45: JAPAN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)