To Gain More Insights into the Neurofibromatosis Treatment Drugs Market, Browse Summary of the Research Report –
Neurofibromatosis is a genetic disorder of the nervous system. It mainly affects growth of nerve cells. It is characterized by the growth of tumors on nerves. Neurofibromatosis is considered as a hereditary disease which mainly occurs in children or it can happen due to mutation (change) of genes. Usually the Neurofibromatosis tumors are benign, but sometimes they can become cancerous. There are three types of neurofibromatosis: Type 1 (NF1), Type 2 (NF2), and Schwannomatosis. All three types show varied symptoms.
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There are three types of Neurofibromatosis; Neurofibromatosis type 1, Neurofibromatosis type 2, and Schwannomatosis. Neurofibromatosis type (NF) 1 is a common monogenic disorder of neurocutaneous tissue growth that arises secondary to mutations in the tumor suppressor gene NF1. It features an autosomal dominant pattern of inheritance. Individuals with NF1 typically have an increased predisposition to a variety of benign and malignant tumors and develop neurofibromas, and axillary and inguinal freckling. Neurofibromatosis 2 (NF2) is less common than NF1. Common signs and symptoms of NF 2 include benign, slow-growing tumors affecting the cranial, spinal, and peripheral nerves, as well as the meninges. KOSELUGO- selumetinib is the only drug available for the treatment of Neurofibromatosis Type-1. Moreover, some drugs such as bevacizumab are also used as off-label for the treatment of Neurofibromatosis
Increasing focus of major players on research and development of novel therapies for treating neurofibromatosis. Moreover, clinical studies are well supported by regulatory authorities which is expected to drive the growth of the global neurofibromatosis treatment drugs market. For instance, in October 2019, SpringWorks Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, initiated the Phase 2b ReNeu clinical trial to evaluate the mirdametinib (formerly PD-0325901), an oral, small molecule designed to inhibit MEK1 and MEK2, in children and adult patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (NF1-PN). In July 2019, the European Commission granted Orphan Drug Designation for SpringWorks Therapeutics’ mirdametinib (formerly PD-0325901), an oral, small molecule inhibitor of MEK1 and MEK2, for the treatment of neurofibromatosis type 1 (NF1).
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Table of Contents
1. Research Objectives and Assumptions
◦ Research Objectives
◦ Assumptions
◦ Abbreviations
2. Market Purview
◦ Report Description
▪ Market Definition and Scope
◦ Executive Summary
▪ Market Snippet, By Drug Type
▪ Market Snippet, By Distribution Channel
▪ Market Snippet, By Region
◦ Coherent Opportunity Map (COM)
3. Market Dynamics, Regulations, and Trends Analysis
◦ Market Dynamics
▪ Drivers
▪ Restraints
▪ Market Opportunities
◦ Impact Analysis
◦ Market Trends
◦ Regulatory Scenario
◦ Reimbursement Scenario
◦ Epidemiology
◦ PEST Analysis
◦ Recent Product Launch
◦ Merger and Acquisition Scenario
Top players in the market
Major Player operating in the global neurofibromatosis treatment drugs market is AstraZeneca Plc.
Research methodology adopted by Coherent Market Insights
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• Primary Research (Trade Surveys and Experts Interviews)
• Desk Research
• Proprietor Data Analytics Model
In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.
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