The Royal Institution for the Advancement of Learning / Mcgill University

Mcgill University

Danuta Radzioch

Samuel David

Ruben Lopez-Vales

Thomas Skinner

Claudine Guilbault

Juan Bautista De Sanctis

John Hanrahan

Asmahan Abu-Arish

Dusan Garic

Methods for enhancing the activity of an ion transporter in a cell and/or treating a channelopathy in a subject based on the administration or use of an effective amount of a fenretinide compound (fenretinide, a fenretinide analog, or a pharmaceutically acceptable salt thereof) and/or zinc are described. The methods are useful for restoring or increasing the cell surface expression of an ion transporter such as the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) that is mutated or defective. The fenretinide compound and/or zinc may be used in combination with ion transporter modulators, e.g., CFTR modulators, to further enhance the cell surface expression and activity of the ion transporter.

Compositions and methods for enhancing ion transporter activity and uses thereof

Compounds, methods, uses, compositions, kits and packages for the prevention and/or treatment of neural injury or a neurodegenerative disease, based on the use of a retinoic acid derivative, such as fenretinide, and/or analogs, derivatives, prodrugs, precursors thereof, and/or salts thereof, are described.

Treatment of neural diseases or conditions

The present invention relates to methods for the treatment of a disease or condition associated with lipid imbalance, comprising (a) decreasing arachidonic acid (AA) levels, (b) increasing docosahexaenoic acid (DHA) levels, (c) increasing DHA/AA ratio or (d) any combination of (a)-(c) in a subject using fenretinide. The invention also relates to diagnostic and screening methods based on the determination of lipid levels.

Method for correcting a lipid imbalance in a subject

Compounds, methods, uses, compositions, kits and packages for the prevention and/or treatment of neural injury or a neurodegenerative disease, based on the use of a retinoic acid derivative, such as fenretinide, and/or analogs, derivatives, pro-drugs, precursors thereof, and/or salts thereof, are described.

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St-Laurent, Canada

Danuta Radzioch