Batten disease is inherited disorder of the nervous system which begins in childhood. It is also called as neuronal ceroid lipofuscinoses (or NCLs). According to Batten Disease Support and Research Association, Batten is ultra-rare, occurring in 1-2 for every 100,000 live births batten disease in U.S. Mostly batten disease is common in Finland, other parts of Northern Europe, North America and Sweden. Till date, there is no treatment available for stopping or reversing the symptoms of batten disease but there are few tests such as urine test or blood test, tissue sampling, measurement of enzyme activity, diagnostic imaging using magnetic resonance imaging (MRI) or computed tomography (CT). As there is huge potential in this industry many companies are vying to plunge into this untapped market by providing solutions for the treatment of batten disease. For instance, disease CLN2 Disease, therapeutic BMN 190, recombinant human tripeptidyl peptidase-1 (rhTPP1), a molecule of BioMarin is currently in phase III clinical trials.
There are four main types of batten diseases
- Infantile NCL: > 2 years of age
- Late infantile NCL: 2 – 4 years of age (the lifespan is 8 to 12 years)
- Juvenile NCL: 5 – 8 years (the lifespan is teens the to early 20s)
- Adult NCL: >40 years (the lifespan varies)
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Batten disease treatment market is expected to deliver positive response due to patient education programs (PEPs), national institutes taking initiatives in research and development of drugs that can help cure the batten disease, a rise in the incidence of batten diseases along with increasing healthcare expenditure are some key drivers of this market. However, factor such as no specific treatment for batten disease treatment is putting adverse impact on this market.
Batten disease treatment market is broadly classified on the basis of therapy and regional prevalence. As of now, there is no treatment for batten disease that can stop but seizures can be used reduced and control this disease. There are some clinical trials & studies being carried out in the North America, Western Europe, and Asia Pacific. Children receiving vitamin supplements resulted slowing of the disease. For patients, there are occupational and physical therapies to retain their functions which so far these treatments have not prevented the final outcome of the disease.
University College London with the help of a service of the U.S. National Institutes of Health has carried out few clinical trials on a mouse and bigger animals for gene therapy, stem cell therapy, enzyme therapy, drug therapy and immune therapy.
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The batten disease treatment market is expected to generate more demand from developed economies comprising North America and Europe. Due to increasing incidence of batten diseases, increasing healthcare expenditure and a presence of the U.S. and Europe-based manufacturers in the region is expected to boost the overall the batten disease treatment market in North America and Europe. However, due to lack of awareness among the manufacturers and patient population Asia-Pacific region is expected to account for the less market share for the overall batten diseases market. Treatment for batten diseases is widely needed globally.
Some of the key market players in batten disease treatment market are:
- BioMarin Pharmaceuticals
- Seneb BioSciences
- ReGenX Biosciences LLC etc.