Metachromatic leukodystrophy (MLD) is an inherited disease that causes fat accumulation in cells. The disease affects the nervous system which produces myelin sheath for insulation and protection of nerves. The fatty deposition, called sulfatides in nerve cells causes the destruction of white matter, which is formed by myelin sheath. It further deteriorates the cognitive functioning of nerves such as an ability to walk, loss of sensation (also known as peripheral neuropathy), seizure, incontinence, inability to speak, paralysis, hearing loss, and blindness. The late infantile form is the common form of metachromatic leukodystrophy that affects in the second year of life and causes loss of speech, gait disturbances and weakness in children. In patients with metachromatic leukodystrophy, the mutation in ARSA gene causes the deficiency of the enzyme, arylsulfatase A, which helps in breakdown of sulfatides. This leads to accumulation of sulfatides in nerve cells. The Shenzhen University is in the process of developing transduced CD34+ hematopoietic stem cell for the treatment of metachromatic leukodystrophy. The University of Minnesota is in the process of developing stem cell transplantation for the treatment of metachromatic leukodystrophy. Some of the companies having the pipeline of metachromatic leukodystrophy include Shire plc, GlaxoSmithKline plc, Zymenex A/S and others.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with the 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, licensing, grants, technology, and others.