Canavan disease is a rare inherited disorder that damages the ability of nerve cells (neurons) in the brain to send and receive messages. Canavan disease is one of a group of genetic disorders, known as leukodystrophies. Leukodystrophies disrupt the growth or maintenance of the myelin sheath, which is the covering that protects nerves and promotes the efficient transmission of nerve impulses. Neonatal/infantile Canavan disease is the most common and most severe form of the condition. Affected infants appear normal for the first few months of life. By the age of three to five months, problems with the infant development become noticeable. These infants usually do not develop motor skills such as turning over, controlling head movement, and sitting without support. Other common features of Canavan disease include weak muscle tone (hypotonia), an unusually large head size (macrocephaly), and irritability. Feeding and swallowing difficulties, seizures, and sleep disturbances may also develop. The mild/juvenile form of Canavan disease is less common. Affected individuals have mildly delayed development of speech and motor skills starting in childhood. These delays may be so mild and nonspecific that they are never recognized as being caused by Canavan disease. The drug candidates in the pipeline of Canavan disease include, but are not limited to, OPC 1 and ALD 101. Some of the companies having drugs in the Canavan disease pipeline include Turing Pharmaceuticals AG and Pfizer 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.