Phosphoglucomutase (PGM 1) Deficiency- Pipeline Insight, 2019
This report can be delivered to the clients within 24 Hours
"Phosphoglucomutase (PGM 1) Deficiency- Pipeline Insight, 2019" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Phosphoglucomutase (PGM 1) Deficiencypipeline landscape is provided which includes the disease overview and Phosphoglucomutase (PGM 1) Deficiencytreatment guidelines. The assessment part of the report embraces, in depth Phosphoglucomutase (PGM 1) Deficiencycommercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phosphoglucomutase (PGM 1) Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Phosphoglucomutase (PGM 1) DeficiencyUnderstanding
Congenital disorders of glycosylation (CDGs) are a group of genetic metabolic diseases that are caused by defects in protein glycosylation. Phosphoglucomutase-1 deficiency (PGM1-CDG) is a special CDG that offers new insights into the highly complex relationship among protein glycosylation, other metabolic pathways, and organ system development, according to Sunnie Yan et al (2016). PGM1 Deficiency is caused by loss of function mutations in the PGM1 gene encoding an enzyme responsible for the interconversion of glucose-6-phosphate to glucose-1-phosphate. Glucose-1-phosphate can be utilized to supply UDP-galactose, a substrate that donates galactose subunits for glycoprotein synthesis. The phenotype of PGM1-CDG comprises a wide range of clinical manifestations, including hypoglycemia, congenital malformations, early-onset dilated cardiomyopathy, growth retardation, hormonal deficiencies, hepatopathy, hemostatic anomalies, and myopathy.
Phosphoglucomutase (PGM 1) DeficiencyPipeline Development Activities
The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Phosphoglucomutase (PGM 1) Deficiencytargeted therapeutics development with respective active and inactive (dormant or discontinued) projects with the appropriate reasons if available. Phosphoglucomutase (PGM 1) Deficiencypipeline report covers only one company. Some of the key players include Cerecor Inc. (CERC-801) etc.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.
Phosphoglucomutase (PGM 1) DeficiencyAnalytical Perspective by DelveInsight
Scope of the report
"Phosphoglucomutase (PGM 1) Deficiency- Pipeline Insight, 2019" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Phosphoglucomutase (PGM 1) Deficiencypipeline landscape is provided which includes the disease overview and Phosphoglucomutase (PGM 1) Deficiencytreatment guidelines. The assessment part of the report embraces, in depth Phosphoglucomutase (PGM 1) Deficiencycommercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Phosphoglucomutase (PGM 1) Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Phosphoglucomutase (PGM 1) DeficiencyUnderstanding
Congenital disorders of glycosylation (CDGs) are a group of genetic metabolic diseases that are caused by defects in protein glycosylation. Phosphoglucomutase-1 deficiency (PGM1-CDG) is a special CDG that offers new insights into the highly complex relationship among protein glycosylation, other metabolic pathways, and organ system development, according to Sunnie Yan et al (2016). PGM1 Deficiency is caused by loss of function mutations in the PGM1 gene encoding an enzyme responsible for the interconversion of glucose-6-phosphate to glucose-1-phosphate. Glucose-1-phosphate can be utilized to supply UDP-galactose, a substrate that donates galactose subunits for glycoprotein synthesis. The phenotype of PGM1-CDG comprises a wide range of clinical manifestations, including hypoglycemia, congenital malformations, early-onset dilated cardiomyopathy, growth retardation, hormonal deficiencies, hepatopathy, hemostatic anomalies, and myopathy.
Phosphoglucomutase (PGM 1) DeficiencyPipeline Development Activities
The report provides insights into different therapeutic candidates in discovery and preclinical, phase 1, phase 2, and phase 3 stage. Drugs under development as a monotherapy or combination therapy are also included. It also analyses key players involved in Phosphoglucomutase (PGM 1) Deficiencytargeted therapeutics development with respective active and inactive (dormant or discontinued) projects with the appropriate reasons if available. Phosphoglucomutase (PGM 1) Deficiencypipeline report covers only one company. Some of the key players include Cerecor Inc. (CERC-801) etc.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university web sites and industry-specific third party sources, etc.
Phosphoglucomutase (PGM 1) DeficiencyAnalytical Perspective by DelveInsight
- In-depth Phosphoglucomutase (PGM 1) Deficiency Commercial Assessment of products
- Phosphoglucomutase (PGM 1) Deficiency Clinical Assessment of products
Scope of the report
- The Phosphoglucomutase (PGM 1) Deficiencyreport provides an overview of therapeutic pipeline activity for Phosphoglucomutase (PGM 1) Deficiencyacross the complete product development cycle including all clinical and non-clinical stages
- It comprises of detailed profiles of Phosphoglucomutase (PGM 1) Deficiencytherapeutic products with key coverage of developmental activities including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product related details
- Detailed Phosphoglucomutase (PGM 1) DeficiencyResearch and Development progress and trial details, results wherever available, are also included in the pipeline study
- Therapeutic assessment of the active pipeline products by development stage, product type, route of administration, molecule type, and MOA type
- Coverage of dormant and discontinued pipeline projects along with the reasons if available across Phosphoglucomutase (PGM 1) Deficiency
- Establish a comprehensive understanding of the current pipeline scenario across Phosphoglucomutase (PGM 1) Deficiencyto formulate effective R&D strategies
- Assess challenges and opportunities that influence Phosphoglucomutase (PGM 1) DeficiencyR&D
- Develop strategic initiatives by understanding the focus areas of leading companies.
- Gather impartial perspective of strategies of the emerging competitors having potentially lucrative portfolio in this space and create effective counter strategies to gain competitive advantage
- Get in detail information of each product with updated information on each project along with key milestones
- Devise Phosphoglucomutase (PGM 1) Deficiencyin licensing and out licensing strategies by identifying prospective partners with progressing projects for Phosphoglucomutase (PGM 1) Deficiencyto enhance and expand business potential and scope
- Our extensive domain knowledge on therapy areas support the clients in decision-making process regarding their therapeutic portfolio by identifying the reason behind the inactive or discontinued drugs
1. REPORT INTRODUCTION
2. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY
2.1. Phosphoglucomutase (PGM 1) Deficiency Disease Overview
2.2. Phosphoglucomutase (PGM 1) Deficiency History
2.3. Phosphoglucomutase (PGM 1) Deficiency Symptoms
2.4. Phosphoglucomutase (PGM 1) Deficiency Causes
2.5. Phosphoglucomutase (PGM 1) Deficiency Pathophysiology
2.6. Phosphoglucomutase (PGM 1) Deficiency Diagnosis
2.6.1. Diagnostic Guidelines
3. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY CURRENT TREATMENT PATTERNS
3.1. Treatment Guidelines
4. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- DELVEINSIGHT’S ANALYTICAL PERSPECTIVE
4.1. In-depth Commercial Assessment
4.1.1. Phosphoglucomutase (PGM 1) Deficiency companies collaborations, Licensing, Acquisition –Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Phosphoglucomutase (PGM 1) Deficiency Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing / Partnering) Analysis
4.1.2.2. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
4.2. Clinical Assessment of Pipeline Drugs
4.2.1. Assessment by Phase of Development
4.2.2. Assessment by Product Type (Mono / Combination)
4.2.2.1. Assessment by Stage and Product Type
4.2.3. Assessment by Route of Administration
4.2.3.1. Assessment by Stage and Route of Administration
4.2.4. Assessment by Molecule Type
4.2.4.1. Assessment by Stage and Molecule Type
4.2.5. Assessment by MOA
4.2.5.1. Assessment by Stage and MOA
5. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY PIPELINE THERAPEUTICS
5.1. Late Stage Products (Phase-III)
5.1.1. Comparative Analysis
5.2. Mid Stage Products (Phase-II)
5.2.1. Comparative Analysis
5.3. Early Stage Products (Phase-I)
5.3.1. Comparative Analysis
5.4. Pre-clinical and Discovery Stage Products
5.4.1. Comparative Analysis
5.5. Inactive Products
6. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY-PRODUCTS ANALYSIS
6.1. Product Profiles
6.1.1. CERC-801- Cerecor Inc.
6.1.1.1. Product Description
6.1.1.1.1. Product Overview
6.1.1.1.2. Mechanism of Action
6.1.1.2. Research and Development
6.1.1.2.1. Clinical Studies
6.1.1.2.1.1. Detailed Study Description
6.1.1.2.1.2. Study Results
6.1.1.2.1.3. Clinical Trials: Tabular View
6.1.1.3. Product Development Activities
6.1.1.3.1. Tabulated Product Summary
6.1.1.3.1.1. General Description Table
To be continued in the report…………………………
7. RECENT TECHNOLOGIES
8. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY KEY COMPANIES
8.1. Cerecor Inc.
9. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY KEY PRODUCTS
9.1. CERC-801
10. DORMANT AND DISCONTINUED PRODUCTS
10.1. Dormant Products
10.1.1. Reasons for being dormant
10.2. Discontinued Products
10.2.1. Reasons for the discontinuation
11. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- UNMET NEEDS
12. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- FUTURE PERSPECTIVES
13. APPENDIX
14. REPORT METHODOLOGY
14.1. Secondary Research
14.2. Expert Panel Validation
2. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY
2.1. Phosphoglucomutase (PGM 1) Deficiency Disease Overview
2.2. Phosphoglucomutase (PGM 1) Deficiency History
2.3. Phosphoglucomutase (PGM 1) Deficiency Symptoms
2.4. Phosphoglucomutase (PGM 1) Deficiency Causes
2.5. Phosphoglucomutase (PGM 1) Deficiency Pathophysiology
2.6. Phosphoglucomutase (PGM 1) Deficiency Diagnosis
2.6.1. Diagnostic Guidelines
3. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY CURRENT TREATMENT PATTERNS
3.1. Treatment Guidelines
4. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- DELVEINSIGHT’S ANALYTICAL PERSPECTIVE
4.1. In-depth Commercial Assessment
4.1.1. Phosphoglucomutase (PGM 1) Deficiency companies collaborations, Licensing, Acquisition –Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Phosphoglucomutase (PGM 1) Deficiency Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing / Partnering) Analysis
4.1.2.2. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
4.2. Clinical Assessment of Pipeline Drugs
4.2.1. Assessment by Phase of Development
4.2.2. Assessment by Product Type (Mono / Combination)
4.2.2.1. Assessment by Stage and Product Type
4.2.3. Assessment by Route of Administration
4.2.3.1. Assessment by Stage and Route of Administration
4.2.4. Assessment by Molecule Type
4.2.4.1. Assessment by Stage and Molecule Type
4.2.5. Assessment by MOA
4.2.5.1. Assessment by Stage and MOA
5. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY PIPELINE THERAPEUTICS
5.1. Late Stage Products (Phase-III)
5.1.1. Comparative Analysis
5.2. Mid Stage Products (Phase-II)
5.2.1. Comparative Analysis
5.3. Early Stage Products (Phase-I)
5.3.1. Comparative Analysis
5.4. Pre-clinical and Discovery Stage Products
5.4.1. Comparative Analysis
5.5. Inactive Products
6. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY-PRODUCTS ANALYSIS
6.1. Product Profiles
6.1.1. CERC-801- Cerecor Inc.
6.1.1.1. Product Description
6.1.1.1.1. Product Overview
6.1.1.1.2. Mechanism of Action
6.1.1.2. Research and Development
6.1.1.2.1. Clinical Studies
6.1.1.2.1.1. Detailed Study Description
6.1.1.2.1.2. Study Results
6.1.1.2.1.3. Clinical Trials: Tabular View
6.1.1.3. Product Development Activities
6.1.1.3.1. Tabulated Product Summary
6.1.1.3.1.1. General Description Table
To be continued in the report…………………………
7. RECENT TECHNOLOGIES
8. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY KEY COMPANIES
8.1. Cerecor Inc.
9. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY KEY PRODUCTS
9.1. CERC-801
10. DORMANT AND DISCONTINUED PRODUCTS
10.1. Dormant Products
10.1.1. Reasons for being dormant
10.2. Discontinued Products
10.2.1. Reasons for the discontinuation
11. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- UNMET NEEDS
12. PHOSPHOGLUCOMUTASE (PGM 1) DEFICIENCY- FUTURE PERSPECTIVES
13. APPENDIX
14. REPORT METHODOLOGY
14.1. Secondary Research
14.2. Expert Panel Validation
LIST OF TABLES
Table 1. Phosphoglucomutase (PGM 1) Deficiency Diagnostic Guidelines
Table 2. Treatment Guidelines
Table 3. Assessment Summary
Table 4. Company-Company Collaborations (Licensing / Partnering) Analysis
Table 5. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
Table 6. Assessment by Phase of Development
Table 7. Assessment by Product Type (Mono / Combination)
Table 8. Assessment by Stage and Product Type
Table 9. Assessment by Route of Administration
Table 10. Assessment by Stage and Route of Administration
Table 11. Assessment by Molecule Type
Table 12. Assessment by Stage and Molecule Type
Table 13. Assessment by MOA
Table 14. Assessment by Stage and MOA
Table 15. Late Stage Products (Phase-III)
Table 16. Mid Stage Products (Phase-II)
Table 17. Early Stage Products (Phase-I)
Table 18. Pre-clinical and Discovery Stage Products
Table 19. Inactive Products
Table 20. Dormant Products
Table 21. Discontinued Products
Table 1. Phosphoglucomutase (PGM 1) Deficiency Diagnostic Guidelines
Table 2. Treatment Guidelines
Table 3. Assessment Summary
Table 4. Company-Company Collaborations (Licensing / Partnering) Analysis
Table 5. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
Table 6. Assessment by Phase of Development
Table 7. Assessment by Product Type (Mono / Combination)
Table 8. Assessment by Stage and Product Type
Table 9. Assessment by Route of Administration
Table 10. Assessment by Stage and Route of Administration
Table 11. Assessment by Molecule Type
Table 12. Assessment by Stage and Molecule Type
Table 13. Assessment by MOA
Table 14. Assessment by Stage and MOA
Table 15. Late Stage Products (Phase-III)
Table 16. Mid Stage Products (Phase-II)
Table 17. Early Stage Products (Phase-I)
Table 18. Pre-clinical and Discovery Stage Products
Table 19. Inactive Products
Table 20. Dormant Products
Table 21. Discontinued Products
LIST OF FIGURES
Figure 1. Disease Overview
Figure 2. History
Figure 3. Symptoms
Figure 4. Causes
Figure 5. Pathophysiology
Figure 6. Diagnostic Guidelines
Figure 7. Treatment Guidelines
Figure 8. Phosphoglucomutase (PGM 1) Deficiency companies collaborations, Licensing, Acquisition –Deal Value Trends
Figure 9. Company-Company Collaborations (Licensing / Partnering) Analysis
Figure 10. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
Figure 11. Assessment by Phase of Development
Figure 12. Assessment by Product Type (Mono / Combination)
Figure 13. Assessment by Stage and Product Type
Figure 14. Assessment by Route of Administration
Figure 15. Assessment by Stage and Route of Administration
Figure 16. Assessment by Molecule Type
Figure 17. Assessment by Stage and Molecule Type
Figure 18. Assessment by MOA
Figure 19. Assessment by Stage and MOA
Figure 20. Late Stage Products (Phase-III)
Figure 21. Mid Stage Products (Phase-II)
Figure 22. Early Stage Products (Phase-I)
Figure 23. Pre-clinical and Discovery Stage Products
Figure 24. Inactive Products
Figure 25. Dormant Products
Figure 26. Discontinued Products
Figure 27. Unmet Needs
KEY COMPANIES
Cerecor
Figure 1. Disease Overview
Figure 2. History
Figure 3. Symptoms
Figure 4. Causes
Figure 5. Pathophysiology
Figure 6. Diagnostic Guidelines
Figure 7. Treatment Guidelines
Figure 8. Phosphoglucomutase (PGM 1) Deficiency companies collaborations, Licensing, Acquisition –Deal Value Trends
Figure 9. Company-Company Collaborations (Licensing / Partnering) Analysis
Figure 10. Phosphoglucomutase (PGM 1) Deficiency Acquisition Analysis
Figure 11. Assessment by Phase of Development
Figure 12. Assessment by Product Type (Mono / Combination)
Figure 13. Assessment by Stage and Product Type
Figure 14. Assessment by Route of Administration
Figure 15. Assessment by Stage and Route of Administration
Figure 16. Assessment by Molecule Type
Figure 17. Assessment by Stage and Molecule Type
Figure 18. Assessment by MOA
Figure 19. Assessment by Stage and MOA
Figure 20. Late Stage Products (Phase-III)
Figure 21. Mid Stage Products (Phase-II)
Figure 22. Early Stage Products (Phase-I)
Figure 23. Pre-clinical and Discovery Stage Products
Figure 24. Inactive Products
Figure 25. Dormant Products
Figure 26. Discontinued Products
Figure 27. Unmet Needs
KEY COMPANIES
Cerecor
