Report Summary: (2019-2026)
Data Bridge Market research presents the Top quality and comprehensive BATTEN DISEASE TREATMENT research report which provides clear insights into market dynamics and prospects the whole market including global production, revenue forecast, value and volume. This report deeply study competitive scenario and evaluated the crucial vendors with regard to product stratification and business strategies.
Market Segment by Companies, this report covers Pfizer Inc, Polaryx, Abeona Therapeutics Inc, Evotec SE, Spark Therapeutics, Inc, Regenxbio Inc, BioMarin, Amicus Therapeutics, Inc, STEMCELL Technologies Inc, Neurogene Inc among others.
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Batten disease is a rare autosomal recessive genetic disorder which mainly affects the nervous system patient. It is also known as neuronal ceroid lipofuscinosis (NCLs). Batten disease occurs due to the deposition of fatty substance called lipopigments in the body’s tissues. Batten disease is one of the most common lysosomal storage disorders. The symptoms include worsening vision, clumsiness, atrophy of brain tissues among others.
According to National Organization for Rare Disorders (NORD), the prevalence of juvenile CLN disease is equal in males and females. In the U.S the occurrence of neuronal ceroid lipofuscinoses is approximately three in 100,000 births and one in 25,000 infants in northern Europe.
Segmentation of the market
- By Types (Congenital NCL, Infantile NCL, Late Infantile NCL and Adult NCL),
- Drugs (Cerliponase Alfa, Valproic Scid and Levetiracetam),
- Therapy (Occupational Therapy and Physical Therapy),
- Treatment (Medication and Supportive Care),
- Route of Administration (Oral and Parenteral),
- Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Online Pharmacy),
- End-Users (Hospitals, Homecare, Specialty Clinics and Others)
Geographical landscape covered in the report
This BATTEN DISEASE TREATMENT report covers a geographical landscape including the region like North America, South America, Asia and Pacific region, Middle East and Africa and Europe. Other vital key regions are also taken into account while developing this report.
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Questions related to the report:
- What does this BATTEN DISEASE TREATMENT market research report offers?
This BATTEN DISEASE TREATMENT report helps you to find about the key industry trends, threats and opportunities. It informs your decision for marketing strategy and planning. The most important its BATTEN DISEASE TREATMENT report helps you to quickly build competitive intelligence in this rapidly changing market.
- Which techniques are utilized to overcome the threats?
Before creating this BATTEN DISEASE TREATMENT report different type of tools and techniques such as SWOT analysis, Porter’s five analysis, Pestle Analysis, Industry chain analysis are utilized which helps the readers to overcome the threats of the market.
Competitive Analysis of the market
The BATTEN DISEASE TREATMENT report tracks the key competitors in the market along with the company profiles which provide market position and basic information of the key competitors. Revenue figure, growth rate and gross margin is also included in the report.
Market Drivers and Restraints
- Increasing prevalence of neurological disorders is driving the market growth
- Increasing global healthcare expenditure is also enhancing this market growth
- Only one drug is approved for Batten disease which is hampering the market growth
- High cost of treatment is another factor hampering this market growth
- Lack of availability of essential services in remote areas is hindering the market growth
Key Developments in the Market
In June 2019, Abeona Therapeutics Inc received the U.S FDA Fast Track designation for ABO-202, one-time gene therapy for the treatment of children with infantile Batten disease. This designation by the U.S FDA is granted to accelerate the review of new compounds that have potential to fulfill the unmet medical needs
In August 2018, Regenxbio Inc announces the pipeline expansion with RGX-181 treating late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease caused by mutation in the tripeptidyl peptidase 1 (TPP1) gene. The current treatment includes palliative care or enzyme replacement therapy where the TPP1 gene is administered into the lateral ventricles. The company submits this drug to the U.S Food and Drug Administration launching a new innovative drug in the market for batten disease treatment
Major Points Covered in Table of Contents:
- Global Batten Disease Treatment Platforms Market Overview
- Economic Impact on Industry
- Market Competition by Manufacturers
- Production, Revenue (Value) by Region
- Supply (Production), Consumption, Export, Import by Regions
- Production, Revenue (Value), Price Trend by Type
- Global Batten Disease Treatment Platforms Market Analysis by Application
- Manufacturing Cost Analysis
- Industrial Chain, Sourcing Strategy and Downstream Buyers
- Marketing Strategy Analysis, Distributors/Traders
- Market Effect Factors Analysis
- Global Batten Disease Treatment Platforms Market Forecast
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This BATTEN DISEASE TREATMENT research report gives the outcome that matters most to the client. The research carried out in this report suggests what is going to be beneficial and how the report will help the business to grow.