Aortic valve calcification is a condition in which calcium deposits form on the aortic valve in the heart. These deposits can cause narrowing at the opening of the aortic valve. This narrowing can become severe enough to reduce blood flow through the aortic valve—a condition called aortic valve stenosis. The inventors have shown that retinoic acid decreases calcification and osteoblast-like phenotype in valvular interstitial cells (VICs). More particularly, RARα activation reduces calcification and osteoblast-like phenotype in VIC. On the contrary, ALDH1A1 inhibition increases calcification and osteoblast-like phenotype in VIC. Thus the results prompt to consider that use or retinoic acid receptor (RAR) agonists would be suitable for the reversing, preventing or delaying calcification of the aortic valve.

A compound for use in the treatment of CNS disorders with sleep disturbances e.g. narcolepsy or Angelman syndrome in a subject, wherein said compound is according to formula (I) or any isomer, tautomer, enantiomer, racemic form or deuterated form thereof, or a pharmaceutically acceptable salt thereof.

A compound for use in the treatment of CNS disorders with sleep disturbances e.g. narcolepsy or Angelman syndrome in a subject, wherein said compound is according to formula (I) or any isomer, tautomer, enantiomer, racemic form or deuterated form thereof, or a pharmaceutically acceptable salt thereof.

The present disclosure relates to the to the transdermal administration of active agents, such as diclofenac and/or CBD and/or THC, and derivatives of these compounds, for the treatment and/or prevention and/or control of chronic pain.

The present disclosure relates to the to the transdermal administration of active agents, such as diclofenac and/or CBD and/or THC, and derivatives of these compounds, for the treatment and/or prevention and/or control of chronic pain.

Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both.

The present invention provides a novel use of a compound having a plasminogen activator inhibitor-1 (PAI-1) inhibitory effect. Specifically, a compound having a PAI-1 inhibitory effect is used as an intranostimulator, an immune checkpoint inhibitor, an inhibitor for exacerbation of tumor cells caused by PD-L1, or an enhancer of immunotherapy for tumors, based on the inhibitory effect of the compound on expression induction of immune checkpoint molecules.

The present invention provides a novel use of a compound having a plasminogen activator inhibitor-1 (PAI-1) inhibitory effect. Specifically, a compound having a PAI-1 inhibitory effect is used as an intranostimulator, an immune checkpoint inhibitor, an inhibitor for exacerbation of tumor cells caused by PD-L1, or an enhancer of immunotherapy for tumors, based on the inhibitory effect of the compound on expression induction of immune checkpoint molecules.

Method of increasing platelet counts in a subject, the method comprising administering to the subject a therapeutically effective amount of a compound that inhibits Biliverdin reductase B (BLVRB) activity by blocking a binding site of BLVRB or a pharmaceutically acceptable salt thereof, wherein the compound does not contain xanthene or acridine moiety is provided.

This invention relates to methods of preparing nanotherapeutic compounds and compositions comprising nanotherapeutic compounds. The nanotherapeutic compounds prepared according to the methods provided herein are useful for the treatment of disease, for example, cancer, in a subject in need thereof.