Globoid cell leukodystrophy, also known as Krabbe disease, is a genetic disorder which is characterized by decreased production of galactocerebrosidase. The symptoms observed during Krabbe disease are fever, vomiting, loss of head control, irritability and excessive crying, seizures, poor coordination of movement or stiffness, muscle spasms, changes in muscle tone, deterioration of motor function, difficulty walking and muscle weakness. There is no cure for Krabbe disease, however, hematopoietic stem cell transplant (HSCT) has demonstrated some positive effects on the disease. Moreover, patient’s quality of life can be improved by anticonvulsant medication to stop seizures, muscle relaxer drugs, physical therapy to help slow deterioration of muscles and occupational therapy. MediciNova Inc. is developing MN-166 (ibudilast) as a novel, orally bioavailable small molecule compound which exerts its effects through several mechanisms to produce its anti-fibrotic and anti-inflammatory activity for the treatment of Krabbe disease. BioXcel Corporation, and Zymenex A/S are some other key players involved in the development of therapeutics for Krabbe disease.
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.