DelveInsight’s, “Spinocerebellar Ataxias – Pipeline Insights, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Spinocerebellar Ataxias pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
- Gait ataxia and incoordination
- Nystagmus/visual problems
- Extrapyramidal signs
- Cognitive impairment in specific SCAs
A phenotype-first approach remains pertinent in molecular diagnosis of rare genetic disorders. Clinicians should also consider genetic testing for primary episodic ataxias (EA). Advances in next-generation sequencing (NGS) have facilitated further insights into the molecular causes of SCA. Exome sequencing identifies less classical phenotype–genotype correlations and detects new mutations in known cerebellar genes.
Advancements in the understanding of pathophysiologic mechanisms facilitate the potential to find new therapeutic targets. Riluzole, a drug licensed for the treatment of ALS, was reconsidered for the treatment of patients with ataxia owing to its ability to inhibit presynaptic glutamate release and activate calcium-activated potassium channels.The anticonvulsant valproic acid has been proposed as a pharmacological treatment in SCA3/MJD for its neuroprotective properties as a pan-histone deacetylase inhibitor. Lithium carbonate was evaluated in 62 ambulatory patients with SCA3/MJD for 48 weeks, but no difference was seen in mean scores of the neurological scale for ataxia. Varenicline is a partial agonist of the α4β2 neuronal nicotinic acetylcholine receptor that is used to aid the cessation of smoking. Varenicline was anecdotally reported to have beneficial effects in patients with ataxia that were taking it as part of a smoking cessation programme. General supportive management options include physiotherapy, occupational therapy and speech therapy. Physiotherapy focused on improving gait, balance, coordination, posture and muscle strength is often recommended.
Spinocerebellar Ataxias Emerging Drugs Chapters
This segment of the Spinocerebellar Ataxias report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Spinocerebellar Ataxias Emerging Drugs
KPS-0373 by Kissei Pharmaceutical
KPS-0373 is an l drug that is a TRH receptor agonist. It has recently completed Phase III trial of clinical study. It was originally developed by Kissei Pharmaceutical but now licensed to Shinogi.
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Table of contents:
1. Report Introduction
2. Spinocerebellar Ataxia
3. Spinocerebellar Ataxia Current Treatment Patterns
4. Spinocerebellar Ataxia- DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Spinocerebellar Ataxia Late Stage Products (Phase-III)
7. Spinocerebellar Ataxia Mid Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Spinocerebellar Ataxia Discontinued Products
13. Spinocerebellar Ataxia Product Profiles
14. Spinocerebellar Ataxia Key Companies
15. Spinocerebellar Ataxia Key Products
16. Dormant and Discontinued Products
17. Spinocerebellar Ataxia Unmet Needs
18. Spinocerebellar Ataxia Future Perspectives
19. Spinocerebellar Ataxia Analyst Review
21. Report Methodology
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