Batten disease is a fatal, inherited disorder of the nervous system that typically begins in the childhood. Early symptoms of Batten disease usually appear between the age of five to 10 years, when parents or physicians may notice a previously normal child has begun to develop vision problems or seizures. In some cases, the early signs are subtle, taking the form of personality and behaviour changes, slow learning, clumsiness, or stumbling. It has been observed that over a period of time, affected children suffer from cognitive impairment, worsening seizures, and progressive loss of sight and motor skills. Eventually, children with Batten disease become blind, bedridden, and demented. Batten disease is often fatal by the late teens or twenties. Though recent improvements in genetic testing have made diagnosing Batten disease much quicker and more reliable. The drug candidates of Batten disease pipeline include, but are not limited to, ABO 202, CLN2 gene therapy and PLX 200.
Some of the companies having drugs in the batten disease pipeline includes Abeona Therapeutics, Inc., Evotec AG, Ionis Pharmaceuticals, Inc. and Spark Therapeutics, Inc.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities including information about clinical results, designations, collaborations, licencing, grants, technology and others.