A61P25/08

TERPENOPHENOLIC COMPOUNDS AND THEIR USE
20230227390 · 2023-07-20 ·

The present disclosure describes terpenophenolic compounds and their use in medicine. The present disclosure further describes perrottetinene-like compounds, the manufacture thereof, formulations containing same and their use in medicine. Such compounds include (1′R, 2′R)-5′-methyl-phenethyl-2′-(prop-1-ene-2-yl)-1′, 2′, 3, 4′-tetrahydro-[1, 1-biphenyl]-2,6-diol (CBD-PET) or (1′R,2′R)-4-(4-hydroxyphenethyl)-5′-methyl-2′-(prop-1-ene-2-yl)-1′, 2′, 3, 4′-tetrahydro-[1, 1′-biphenyl]-2,6-diol (CBD-PET-OH).

TETRAHYDROCANNABINOL DERIVATIVES, PREPARATION METHOD THEREOF AND USE THEREOF

Tetrahydrocannabinol derivatives and medical use thereof, in particular to the compounds represented by general formula (I), or stereoisomers, solvates, metabolites, pharmaceutically acceptable salts or cocrystals thereof, wherein the definitions of substituents in general formula (I) are the same as those in the description

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Tizanidine therapy system

An object of the present invention is to provide a method for the treatment of a patient suffering from spasticity comprising administering to said patient a modified release dosage form comprising tizanidine or a pharmaceutically acceptable salt thereof. The present invention relates to a method of administering a transdermal patch preparation comprising tizanidine or a pharmaceutically acceptable salt thereof to a subject in need thereof, wherein the transdermal patch preparation releases about 4 mg to about 36 mg of tizanidine or a pharmaceutically acceptable salt thereof for at least about 24 hours.

METHODS AND COMPOSITIONS FOR TREATING EPILEPSY

The present disclosure provides methods and compositions relating to gene therapy for treating epilepsy, such as a temporal lobe epilepsy, in a subject in need thereof by targeting Grik2 mRNA. In particular, the present disclosure provides inhibitory RNA constructs capable of inhibiting expression of GluK2 protein and inhibiting epileptiform discharges in hyperexcitable neuronal circuit.

METHODS AND COMPOSITIONS FOR TREATING EPILEPSY

The present disclosure provides methods and compositions relating to gene therapy for treating epilepsy, such as a temporal lobe epilepsy, in a subject in need thereof by targeting Grik2 mRNA. In particular, the present disclosure provides inhibitory RNA constructs capable of inhibiting expression of GluK2 protein and inhibiting epileptiform discharges in hyperexcitable neuronal circuit.

L-serine compositions, methods and uses for treating neurodegenerative diseases and disorders

L-serine, L-serine precursors, L-serine derivatives and L-serine conjugates for treatment, amelioration and/or prevention of protein aggregation/tangles/plaques and diseases associated with protein aggregation/tangles/plaques. In particular, treatments and uses for L-serine, L-serine precursors, L-serine derivatives and L-serine conjugates include Alzheimer's disease (AD), Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), and Huntington disease (HD).

Use of cannabinoids in the treatment of epilepsy

The present disclosure relates to the use of cannabidiol (CBD) for the reduction of total convulsive seizure frequency in the treatment of “treatment-resistant epilepsy” (TRE). In particular, the disclosure relates to the use of CBD of treating TRE when the TRE is Dravet syndrome; myoclonic absence seizures or febrile infection related epilepsy syndrome (FIRES). The disclosure further relates to the use of CBD in combination with one or more anti-epileptic drugs (AEDs).

Methods for modulating RNA splicing

In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise an intronic REMS, and the use of a compound described herein to modulate protein produced from such altered endogenous genes.

Ganaxolone for use in treating tuberous sclerosis complex

The disclosure to methods for treating tuberous sclerosis complex or tuberous sclerosis complex-related epilepsy, comprising administering to a subject in need thereof a therapeutically effective amount of a pharmaceutically acceptable pregnenolone neurosteroid, such as ganaxolone, to reduce one or more symptoms of tuberous sclerosis complex or tuberous sclerosis complex-related epilepsy.

Ganaxolone for use in treating tuberous sclerosis complex

The disclosure to methods for treating tuberous sclerosis complex or tuberous sclerosis complex-related epilepsy, comprising administering to a subject in need thereof a therapeutically effective amount of a pharmaceutically acceptable pregnenolone neurosteroid, such as ganaxolone, to reduce one or more symptoms of tuberous sclerosis complex or tuberous sclerosis complex-related epilepsy.