Treatment of Charcot-Marie-Tooth disease

Patent number:

PCT/ES2022/070387; 202130576

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The present invention discloses a pharmaceutical composition for the treatment of Charcot-Marie-Tooth disease comprising florfenicol, preferably being alterations in GDAP1 and the symptoms of the disease motor deficiencies, sensory loss, optical deficiencies, diaphragmatic paralysis and/or paralysis of the vocal cords the cause of said disease.

Countries:

Spain

Regions:

Community of Madrid

Centers:

UNIVERSIDAD AUTONOMA DE MADRID, CIBERNED CENTRO DE INVESTIG BIOMEDICA EN RED ENFERMEDADES NEURODEGENERATIVAS, HOSPITAL SANT JOAN DE DEU

Other entities:

Sectors:

Health

Subsectors:

TRL Level:

TRL 4 – technology validated in lab

BRL Level:

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Sustainable Development Goal:

SDG03: Good health and well-being

Applications

Pharmaceutical composition for the treatment of the symptoms of Charcot-Marie-Tooth (CMT) disease, a neuropathy for which there are currently no effective treatments. The group studies effective therapeutic strategies in a pre-clinical mouse model of Charcot-Marie-tooth disease. It has been showed for preventing the overproduction of reactive oxygen species (ROS) in the mitochondria with florfenicol to prevent the development of symptoms and motor deficits associated with CMT disease in a preclinical mouse model. It can be applied in the field of medicine, for the prevention and treatment of Charcot-Marie-Tooth disease.

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