Overview of the drug development for Rett syndrome
Rett syndrome is one of the rare genetic neurological disorders. It majorly affects the development of the brain. It further leads to the inability of a person to use muscles for body movements and speech. It can also result in seizures and intellectual disability. Long-time complications of Rett syndrome can result in difficulty in eating, constipation, unusual sleep patterns, and muscle and joint problems. As a result, with increasing incidences of the syndrome, the need for drug development for Rett syndrome is expected to increase considerably in the next few years.
According to this pipeline analysis report, most of the drug molecules in the pipeline are being developed for Rett syndrome. 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 Rett syndrome. 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 –
- Amicus Therapeutics
- AMO PHARMA
- Amorsa Therapeutics
- Biohaven Pharmaceutical
Therapeutic assessment of the drug development for Rett syndrome by route of administration
- Oral
- Subcutaneous
- Intrathecal
The oral route of administration (ROA) involves the application of the drug directly into the mouth cavity, which will have a more direct effect on the target cells.
Therapeutic assessment of the drug development for Rett syndrome by therapy
- Monotherapy
According to this pipeline analysis report, all the molecules that are currently in the drug development for Rett syndrome 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 Rett syndrome?
- What are the companies that are currently involved in the development of drug molecules for Rett syndrome?
- 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
Technavio also offers customization on reports based on specific client requirement.