Krabbe disease treatment companies focus on developing therapies and interventions for individuals affected by Krabbe disease, a rare genetic disorder that affects the nervous system. These companies contribute to rare disease research by providing treatments that aim to slow down disease progression and improve the quality of life for individuals with Krabbe disease.
Dec 2023
Several biotech companies like Bluebird bio and Orchard Therapeutics are actively developing gene therapy candidates for Krabbe disease with promising preclinical and early clinical trial results.These therapies aim to address the underlying genetic cause of Krabbe disease, offering potential for long-term remission or cure.Companies like Shire (now part of Takeda) and BioMarin Pharmaceutical Inc. continue to optimize their existing ERT products for Krabbe disease, focusing on improved efficacy and delivery methods.ERT remains a crucial supportive therapy for managing symptoms and slowing disease progression, particularly in infantile Krabbe patients.Research institutions and clinical trials are investigating HSCT as a potential treatment option for Krabbe disease, particularly for late-onset and symptomatic patients.HSCT holds promise for long-term benefits, but research is ongoing to optimize timing, patient selection, and conditioning regimens for better outcomes.
List of Krabbe disease treatment Key companies in the market
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