Spinal Muscular Atrophy - A Pipeline Analysis Report
Published: Jul 2018 | Pages: 109 | SKU: IRTNTR23329 |
Overview of the drug development for spinal muscular atrophy
Spinal Muscular Atrophy (SMA) is a genetic disorder that results in the weakening of the muscles and affects muscle movement. It is common in infants, and it progresses with time. Infants with SMA have a breakdown of nerve cells in the brain and the spinal cord, due to which the brain stops sending control signals that control the muscle movement to the spinal cord. The muscles shrink and become weak, making it tough for the patients to control the head movement, sit without any external support and walk. People with a family history of SMA are at higher risks of developing the disease than the ones with no family history of SMA. The disease develops in the people with two fault copies of survival or motor neuron 1 (SMN1) gene. The people with only one fault copy of SMN1 gene are termed as the carriers of SMA. The disease does not develop in the carriers, and the profitability of the disease development in their offspring is higher. As per the data presented by the SMA Foundation, the incidence of SMA across all genders, races, and ethnic backgrounds is estimated to be 1 in every 10,000 children born. As a result, with the rising incidences of the disease, the drug development for spinal muscular atrophy is expected to rise during the next few years.
According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for spinal muscular atrophy (SMA). Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.
Companies covered
This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of spinal muscular atrophy. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug molecules discontinued by companies.
Some of the companies covered in this pipeline analysis report are –
- CYTOKINETICS
- Novartis
- Scholar Rock
- Voyager Therapeutics
- Recursion Pharmaceuticals
Therapeutic assessment of the drug development for spinal muscular atrophy by route of administration
- Oral
- Intravenous
- Intrathecal
The oral route of administration (ROA) involves the application of the drug directly through the mouth cavity, which will have a more direct effect on the target cells.
Therapeutic assessment of the drug development for spinal muscular atrophy by therapy
- Monotherapy
According to this pipeline analysis report, most of the molecules that are currently in the drug development for spinal muscular atrophy are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.
Key questions answered in the report include
- What are the drug molecules in the various development stages for spinal muscular atrophy?
- What are the companies that are currently involved in the development of drug molecules for spinal muscular atrophy?
- Insight into discontinued/inactive molecules with appropriate reasoning?
- What are the major regulatory authorities approving drugs in various regions?
- Detailed profiling of each active molecule
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PART 01: EXECUTIVE SUMMARY
PART 02: SCOPE OF THE REPORT
PART 03: RESEARCH METHODOLOGY
PART 04: INTRODUCTION
PART 05: MAJOR REGULATORY AUTHORITIES
- US
- Europe
- China
PART 06: PIPELINE LANDSCAPE
PART 07: COMPARATIVE ANALYSIS
- Discovery stage molecules
- Pre-clinical stage molecules
- Inactive and discontinued molecules
PART 08: INDICATION ANALYSIS
PART 09: THERAPEUTIC ASSESSMENT (THERAPY BASED)
PART 10: THERAPEUTIC ASSESSMENT (ROA BASED)
PART 11: THERAPEUTIC ASSESSMENT BY TARGET
PART 12: KEY COMPANIES
- Active companies: Category and parameters
PART 13: APPENDIX
- List of abbreviations
Research Framework
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INFORMATION SOURCES
Primary sources
- Manufacturers and suppliers
- Channel partners
- Industry experts
- Strategic decision makers
Secondary sources
- Industry journals and periodicals
- Government data
- Financial reports of key industry players
- Historical data
- Press releases
DATA ANALYSIS
Data Synthesis
- Collation of data
- Estimation of key figures
- Analysis of derived insights
Data Validation
- Triangulation with data models
- Reference against proprietary databases
- Corroboration with industry experts
REPORT WRITING
Qualitative
- Market drivers
- Market challenges
- Market trends
- Five forces analysis
Quantitative
- Market size and forecast
- Market segmentation
- Geographical insights
- Competitive landscape
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- What are the latest trends influencing the growth of this market?
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