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Globoid Cell Leukodystrophy (Krabbe Disease) is a rare autosomal recessive genetic disorder caused due to a lysosomal enzyme, galactosylceramidase that results in severe neurological condition due to loss of myelin in the nervous system. The disease is also characterized by the detection of abnormal cells in the brain known as globoid cells that have more than one nucleus. The most common infantile form of Krabbe disease begins at the age of one showing symptoms such as irritability, muscle weakness, blindness, deafness, episodes of fever, stiff posture, and delayed mental and physical development.
Currently, there is no specific treatment for globoid cell leukodystrophy (Krabbe Disease). Most treatments for Krabbe disease are supportive. However, hematopoietic stem cell transplant is found to be an effective therapy in patients who have been diagnosed before or at birth. Additionally, the development of a new MS-based biomarker for early and sensitive detection of Krabbe disease from blood is under clinical investigation.
Owing to the rising prevalence of the disease many pharmaceutical companies and research institutes are focusing on studying and developing novel therapy for globoid cell leukodystrophy (Krabbe Disease). For instance; MGTA-456 which is being studied by Magenta Therapeutics is currently in a phase-2 clinical trial for the study of safety, tolerability, and efficacy of MGTA-456 for treatment of patients with globoid cell leukodystrophy (Krabbe Disease).
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At present, around 64% of the pipeline candidates for globoid cell leukodystrophy (Krabbe Disease) are in the phase-2 clinical study. Majority of the studies are sponsored by academic and research universities.
The report on ‘Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline Review, 2019’ provides a comprehensive overview of the drugs that are in the R&D pipeline by indication or molecule for Globoid Cell Leukodystrophy (Krabbe Disease). The report provides a thorough analysis of the distribution of the pipeline products by clinical trial stage, indication, company, therapy area and details such as clinical trial stage, sponsor, description on every product in the pipeline. Products in the preclinical and clinical stage along with dormant & discontinued pipeline candidates are included in the report. The report also covers additional insights such as epidemiology overview and current market scenario for Globoid Cell Leukodystrophy (Krabbe Disease).
The report on ‘Globoid Cell Leukodystrophy (Krabbe Disease) – Pipeline Review, 2019’, which is built by following a robust research methodology involving primary interviews and desk research, provides a complete overview of the R&D activity and pipeline products to assist companies in developing growth strategies and identifying emerging players.
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