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Batten disease is a rare, inherent, and fatal autosomal recessive neural disorder that begins in the childhood but may take a few years to show symptoms. Batten disease is one of the approximately 50 diseases that fall under the category of lysosome shortage disorders. The batten disease also referred as Neuronal ceroid Lipofuscinoses (NCLs), which is a class of life-limiting genetic neurogenerative diseases that are caused due to abnormality of genes resulting in their inability to synthesize required protein. This disease is characterized by malfunctioning of body, which usually appears around the age of 2-10 with symptoms, such as seizures or gradual onset of vision problem. The early signs may show subtle changes in behavior and learning, however over time, the child suffers from mental impairment, progressive loss of sight, speech and motor reactions, worsening seizures, eventually leading to blindness dementia, and finally death. These disease is diagnosed with the help of certain tests, such as blood or urine tests, skin or tissue dampening, brain scans, and imaging techniques, which includes, computed tomography (CT) or magnetic resonance imaging (MRI). The batten disease is characterized into four types as follows:

  • Infantile NCL: more than 2 years
  • Late Infantile NCL: 2-4 years (Life span of child varies between 8-12 years)
  • Juvenile NCL: 5-8 years (Life span varies between teens to early 20s)
  • Adult NCL: more than 40 years (Variable lifespan)

European countries facing rising prevalence and incidence of batten disease

According to the Centers for Disease Control and Prevention (CDC), Batten disease is observed in 2-4 per 100,000 live births in the United States on an average. The Batten disease although is rare but has high incidence rates in Finland, Sweden, and other parts of the Europe as compared to rest of the world. According to the Batten Disease Support and Research Association, the batten disease has often been encountered in more than one children of the same family. The Daily News and Analysis article published in June 2016 suggests that, over 20 million children in India suffer from rare diseases, having expensive treatments. This creates opportunities for the sustaining as well as upcoming market players by understanding the potential held by the global batten disease treatment market.

The global batten disease treatment market is segmented on the basis of therapy and geographical regions.

On the basis of therapy, the global batten disease treatment market is segmented as

  • Occupational Therapies
  • Physical Therapies

As of now, there are no established drugs and treatments to cure and prevent the disease. However, the severity of disease can be reduced and controlled by certain therapies. Few clinical trials and studies are being carried out in the North America, Western Europe, and Asia Pacific to identify therapies that would help reducing the disease outcome.

Attempts by collaborations of research bodies for introduction to reliable treatment options for batten disease giving hopes to commoners

The batten disease treatment market is not dense on the global level, however the developed economies of North America and Europe are expected to contribute to the growth of the market. This attributes to the increasing incidence of batten disease and high prevalence rates in American as well as European countries. For instance, a joint study between Baylor College of Medicine, Texas Children’s Hospital, and King’s College London have discovered a treatment that has helped to improve the neurological symptoms in the mouse model of juvenile batten disease. This research held a novel approach of counteracting the accumulation of the cellular waste in Batten disease by acting on TFEB, which is a master transcription factor that stimulates the production of lysosomes by the cell body and directs cellular apoptosis. This has brought a ray of hope to patients and families affected by battens disease.

Improper treatment facility for batten disease responsible to attract the attention of various players to enter the untapped potential batten disease treatment market

The rising incidence of the batten disease without effective treatment option for the prevention and cure of the disease is the primary driver for batten disease market, due to which several companies are vying to enter into the untapped global batten disease treatment market. However, the lack of awareness among manufacturers and patient population as well as unavailability of treatment for this disease are greatly hampering the growth of the global batten disease treatment market.

Although, the global batten disease treatment market is very scarce, few key players offering cell regeneration therapies and few other forms of medication include Seneb BioSciences, Inc., BioMarin Pharmaceuticals and ReGenX Biosciences LLC. The U.S. FDA has recently approved BioMarin’s first ever drug to treat batten disease known as Brineura in 2017.


Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • 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.

Data Triangulation Methodology | Coherent Market Insights

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.

Market Analysis | Coherent Market Insights

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users