A recombinant adeno-associated virus (rAAV) comprising an AAVhu68 capsid and a vector genome comprising a lysosomal beta-galactosidase gene (for example, galactosidase beta 1 gene, GBL1) is provided (i.e., rAAVhu68.GBL1). Also provided a composition containing an effective amount of rAAVhu68.GBL1 to ameliorate symptoms of GM1 gangliosidosis, including, e.g., increased average life span, decreased need for feeding tube, reduction in seizure incidence and frequency, reduction in progression towards neurocognitive decline and/or improvement in neurocognitive development.

The present invention relates to a cannabinoid containing oral solution. Preferably the cannabinoid is cannabidiol (CBD), cannabidivarin (CBDV) or cannabidiol-C4 (CBD-C4). More preferably the CBD, CBDV or CBD-C4 is present at a concentration of between 25 and 75 mg/ml. More preferably still the CBD, CBDV or CBD-C4 is present at a concentration of 50 mg/ml. In a further embodiment the oral solution is formulated with one or more edible oils. Preferably the edible oil is sesame oil. It is a preferred embodiment of the invention that the oral formulation comprises a low concentration of ethanol.

The present invention relates to compositions and methods of treatment and/or prevention of diseases and conditions associated with amyloid-beta associated diseases. In particular, the present invention relates to compositions comprising propionic acid.

The present invention relates to compositions and methods of treatment and/or prevention of diseases and conditions associated with amyloid-beta associated diseases. In particular, the present invention relates to compositions comprising propionic acid.

Agarophyton chilensis extract, enriched with free fatty acids and PPARγ modulators, comprising palmitic acid, stearic acid, myristic acid, oleic acid, and 8-hydroxyeicosatetraenoic acid (8-HETE). Method for obtaining the extract. Nutraceutical composition, comprising the extract enriched with free fatty acids and PPARγ modulators, wherein said extract is useful for treating or preventing health problems, which require neuroprotection, wherein said neuroprotection involves activation of PPARγ receptors.

Agarophyton chilensis extract, enriched with free fatty acids and PPARγ modulators, comprising palmitic acid, stearic acid, myristic acid, oleic acid, and 8-hydroxyeicosatetraenoic acid (8-HETE). Method for obtaining the extract. Nutraceutical composition, comprising the extract enriched with free fatty acids and PPARγ modulators, wherein said extract is useful for treating or preventing health problems, which require neuroprotection, wherein said neuroprotection involves activation of PPARγ receptors.

The present disclosure relates to transcription cassettes comprising nucleic acids encoding RuvBL1 and/or RuvBL2 and the use of said vectors in gene therapy for the treatment of neurodegenerative diseases that result from expression of polymorphic repeat expansions of the GGGGCC (SEQ ID NO: 5) hexanucleotide-repeat sequence in the first intron of the C9ORF72 gene; pharmaceutical compositions comprising said vectors and including uses and methods to treat neurodegenerative diseases.

The present disclosure relates to transcription cassettes comprising nucleic acids encoding RuvBL1 and/or RuvBL2 and the use of said vectors in gene therapy for the treatment of neurodegenerative diseases that result from expression of polymorphic repeat expansions of the GGGGCC (SEQ ID NO: 5) hexanucleotide-repeat sequence in the first intron of the C9ORF72 gene; pharmaceutical compositions comprising said vectors and including uses and methods to treat neurodegenerative diseases.

The present disclosure features useful compositions and methods to treat trinucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with OGG1 activity.

The present disclosure features useful compositions and methods to treat trinucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with OGG1 activity.