Increasing tfr1 palmitoylation as a novel therapeutic strategy for neurodegeneration with brain iron accumulation

The present invention relates to methods and pharmaceutical compositions for the treatment of neurodegeneration with brain iron accumulation (NBIA). In particular, the present invention relates to a method of treating neurodegeneration with brain iron accumulation comprising administering to the subject a therapeutically effective amount of a drug increasing TfR1 palmitoylation.

Keywords: Neurology - Neurodegeneration - Small molecule - Brain Iron Accumulation
Patent Application number: EP16306740.8
Inventors:
Agnès ROTIGAntony DRECOURT
Publications:
Am J Hum Genet. 2018 Feb 1;102(2):266-277. doi: 10.1016/j.ajhg.2018.01.003.

Reference:

BIO16340-T1

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Anne COCHI
Anne COCHI
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Patent filling date: 20-12-2016
Rare disease: Yes
Second indication: Yes

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