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It has been observed that spinocerebellar ataxia (SCA) is a hereditary disease caused due to mutations in the ATXN1 gene. Mutation in ATXN1 gene can lead to degenerative changes in the cerebellum and spinal cord. A person affected by the disease usually inherits the altered gene from an affected parent. The gene is passed from one generation to the next. According to the NIH, one to two per 100,000 people worldwide are affected by spinocerebellar ataxia. Spinocerebellar ataxia type 3, also called the Machado-Joseph disease (MJD), is an inherited form of ataxia that is characterized by the progressive clumsiness in the arms and legs. MJD is usually classified in three types including Type I, Type II, and Type III. Type I usually starts at the age of 10-30 years with faster progression and more dystonia and rigidity than ataxia. Type II is the most common type of MJD. It starts between the age of about 20 and 50 years and has an intermediate rate of progression. Type III starts at the age of 40-70 years and progresses relatively slowly. Consequently, the rising incidences of the condition are further likely to boost the drug development for spinocerebellar ataxia in the next few years.
According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for spinocerebellar ataxia type 3. 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.
This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of spinocerebellar ataxia type 3. 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.
PART 01: EXECUTIVE SUMMARY
PART 02: SCOPE OF THE REPORT
PART 03: RESEARCH METHODOLOGY
PART 04: INTRODUCTION
PART 05: MAJOR REGULATORY AUTHORITIES
PART 06: PIPELINE LANDSCAPE
PART 07: COMPARATIVE ANALYSIS
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
PART 13: APPENDIX
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