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.
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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.
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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.
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