The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

The present invention provides, in part, compounds of Formula I: or an N-oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: X.sup.1, X.sup.2, R.sup.1, R.sup.2, m and n are as described herein; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds, N-oxides, or salts, and their uses for treating M4-mediated (or M4-associated) disorders including, e.g., Alzheimer's Disease, Parkinson's Disease, schizophrenia (e.g., its cognitive and negative symptoms), pain, addiction, and a sleep disorder.

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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).

The present invention relates to a transmucosal administration system to administer quinones, benzoquinones, and especially 1,4-benzoquinones, via the oromucosal route.

Provided are methods of treating refractory seizures in a human subject, e.g., a child, that include administering an effective amount of cannabidiol (CBD), e.g., in a transdermal gel.

Provided are methods of treating refractory seizures in a human subject, e.g., a child, that include administering an effective amount of cannabidiol (CBD), e.g., in a transdermal gel.

The present invention relates to the use of cannabidiol (CBD) in the treatment of focal seizures. In one embodiment the patients suffering from focal seizures are children and young adults. CBD appears particularly effective in reducing focal seizures in patients suffering with etiologies that include: Lennox-Gastaut Syndrome; Tuberous Sclerosis Complex; Dravet Syndrome; CDKLS; Neuronal ceroid lipofuscinoses (NCL); febrile infection related epilepsy syndrome (FIRES); Aicardi syndrome and brain abnormalities in comparison to other seizure types. Significantly CBD additionally is very effective in the reduction of a sub-type of focal seizures, focal seizures with impairment.

The invention relates to Spiro derivatives, to the use of said derivatives in treating diseases and conditions mediated by modulation of voltage-gated sodium channels, to compositions containing said derivatives and processes for their preparation.

The present disclosure relates to the use of cannabidiol (CBD) for the treatment of seizures associated with Aicardi Syndrome. In one embodiment the seizures associated with Aicardi Syndrome are convulsive seizures; focal seizures with impairment or infantile spasm. The disclosure further relates to the use of CBD in combination with one or more anti-epileptic drugs (AEDs).

Novel hydrogen isotope-enriched 1,2,4-oxadiazole benzoic acid compounds, methods of their use and pharmaceutical compositions thereof are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination.