Disclosed are improved methods for manufacturing large-scale populations of robust, highly pure, and functional T regulatory cells (Tregs). Also disclosed are expanded Treg populations, cryopreserved Treg populations and methods and uses of these cells in compositions formulated for treating one or more mammalian diseases, including, for example, treatment, prophylaxis, and/or amelioration of one or more symptoms of a human neurodegenerative disorder. In particular, the compositions and methods provided herein find clinical use in the treatment and amelioration of one or more symptoms of amyotrophic lateral sclerosis (ALS), Alzheimer's disease, and other neurological diseases and disorders.

Disclosed are improved methods for manufacturing large-scale populations of robust, highly pure, and functional T regulatory cells (Tregs). Also disclosed are expanded Treg populations, cryopreserved Treg populations and methods and uses of these cells in compositions formulated for treating one or more mammalian diseases, including, for example, treatment, prophylaxis, and/or amelioration of one or more symptoms of a human neurodegenerative disorder. In particular, the compositions and methods provided herein find clinical use in the treatment and amelioration of one or more symptoms of amyotrophic lateral sclerosis (ALS), Alzheimer's disease, and other neurological diseases and disorders.

The present disclosure provides methods of treating, preventing, or delaying the onset of a late-onset neurodegenerative disease, e.g., Niemann-Pick type C, or a symptom thereof in a subject in need thereof, comprising administering a therapeutically effective amount of acetyl-leucine to the subject, wherein the subject is heterozygous NPC1 gene mutation carrier.

The present disclosure provides methods of treating, preventing, or delaying the onset of a late-onset neurodegenerative disease, e.g., Niemann-Pick type C, or a symptom thereof in a subject in need thereof, comprising administering a therapeutically effective amount of acetyl-leucine to the subject, wherein the subject is heterozygous NPC1 gene mutation carrier.

The present invention relates to solid pharmaceutical compositions comprising N-[1-(5-cyano-pyridin-2−ylmethyl)-1H-pyrazol-3-yl]-2-[4-(1-trifluoromethyl-cyclopropyl)-phenyl]-acetamide or pharmaceutically acceptable salt thereof. The invention further relates to methods for manufacturing said compositions and their uses for the treatment or prevention of diseases and disorders linked to T-type calcium channels such as epilepsy.

The present invention relates to a biotin moiety-conjugated polypeptide and a pharmaceutical composition for oral administration comprising the same, wherein the polypeptide according to the present invention has an excellent in vivo oral bioavailability.

The present invention relates to a biotin moiety-conjugated polypeptide and a pharmaceutical composition for oral administration comprising the same, wherein the polypeptide according to the present invention has an excellent in vivo oral bioavailability.

Compounds are provided having the structure of Formula (I) or a pharmaceutically acceptable isomer, racemate, hydrate, solvate, isotope, or salt thereof, wherein R.sup.1, R.sup.2, X.sup.1, X.sup.2, Y.sup.1, and Y.sup.2 are as defined herein. Such compounds function as thyromimetics and have utility for treating diseases such as neurodegenerative disorders and fibrotic diseases. Pharmaceutical compositions containing such compounds are also provided, as are methods of their use and preparation.

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The present invention relates to a method or a composition for treating neurodegenerative diseases, and particularly, the present invention relates to a method for treating neurodegenerative diseases of the cerebellum, comprising administering a composition comprising stem cells as an active ingredient to a patient having a neurodegenerative disease of the cerebellum, or to a composition for treating neurodegenerative diseases of the cerebellum comprising mesenchymal stem cells as an active ingredient. The treatment method or the treatment composition using mesenchymal stem cells, according to the present invention, has remarkable effects in reducing neuroinflammation, inhibiting M1 microglia, activating M2 microglia, inhibiting apoptosis of Purkinje cells, inhibiting death of neurons, improving motor ability, and the like, and therefore may be effectively utilized in alleviating and treating neurodegenerative diseases including cerebellar ataxia and multiple system atrophy.

The present invention relates to a method or a composition for treating neurodegenerative diseases, and particularly, the present invention relates to a method for treating neurodegenerative diseases of the cerebellum, comprising administering a composition comprising stem cells as an active ingredient to a patient having a neurodegenerative disease of the cerebellum, or to a composition for treating neurodegenerative diseases of the cerebellum comprising mesenchymal stem cells as an active ingredient. The treatment method or the treatment composition using mesenchymal stem cells, according to the present invention, has remarkable effects in reducing neuroinflammation, inhibiting M1 microglia, activating M2 microglia, inhibiting apoptosis of Purkinje cells, inhibiting death of neurons, improving motor ability, and the like, and therefore may be effectively utilized in alleviating and treating neurodegenerative diseases including cerebellar ataxia and multiple system atrophy.