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Back Temsirolimus, as a new treatment for X-adrenoleukodystrophy

Temsirolimus, as a new treatment for X-adrenoleukodystrophy

Temsirolimus, as a new treatment for X-adrenoleukodystrophy

We propose using rapamycin-related mTOR inhibitors as a potential therapeutic approach for X-linked adrenoleukodystrophy (X-ALD), which is a neurometabolic disease characterized the loss of function of the peroxisomal transporter ABCD1 resulting in the accumulation of very-long chain fatty acids (VLCFA).
 

INDUSTRIAL PROPERTY RIGHTS

A European patent application (EP14382353.2) has been filed.
 

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