Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a ceroid lipofuscinosis neuronal 3 (CLN3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for CLN3 gene therapy of the neuronal ceroid lipofuscinosis or CLN3-Batten Discase.

Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a ceroid lipofuscinosis neuronal 3 (CLN3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for CLN3 gene therapy of the neuronal ceroid lipofuscinosis or CLN3-Batten Disease.

Abstract: The present disclosure relates to methods of treating conditions associated with a need for Merlin protein, for example due to a defective Neurofibromin 2/Merlin (NF2) gene as in neurofibromatosis type 2 (NF2). In particular. the disclosure provides gene therapy vectors to specifically treat loss of expression of the Merlin protein or reduced Merlin protein levels.

Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a neuronal ceroid lipofuscinosis neuronal 6 (CLN6) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for CLN6 gene therapy of the neuronal ceroid lipofuscinosis or CLN6-Batten Disease.

Abstract: The present disclosure relates to gene therapy methods of preserving photoreceptors and/or inhibiting or preventing retinal degeneration in Batten disease patients, including recombinant adeno-associated vims (rAAV) delivery of a neuronal ceroid lipofuscinosis neuronal 6 (CLN6) polynucleotide.

Abstract: The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a ceroid lipofuscinosis neuronal 3 (CLN3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for CLN3 gene therapy of the neuronal ceroid lipofuscinosis or CLN3-Batten Disease.

Abstract: Solubilizers prepared by polymerizing: (i) at least one first monomer of the general formula (I): wherein R1 and R2 each independently represent H or CH3, R3 represents a C6-C10-aryl or C7-C12-aralkyl which may be unsubstituted or substituted with one or more C1-C9-alkyl and/or C1-C5-alkoxy substituents, and n represents an integer from 0 to 100; and (ii) at least one second monomer selected from the group consisting of N-vinylamides, N-vinyllactams, N-vinylimines, N-vinylamines having 2 to 15 carbon atoms, and mixtures thereof; and methods of solubilizing active ingredients by combining them with such a solubilizer.

Abstract: A solid oral pharmaceutical dosage form of a sympathomimetic with impeded extractability of the sympathomimetic, comprising I) a sympathomimetic (component I) II) an excipient mixture (component II) composed of a) 5 to 50% by weight of hydroxyalkylcelluloses or alkylcelluloses or mixtures thereof b) 5 to 70% by weight of xanthan c) 5 to 50% by weight of an alkaline earth metal salt of a C10-C30 fatty acid d) 10 to 70% by weight of an alkali metal or alkaline earth metal salt of a mineral acid, of a sulfonic acid or of a C2-C6 carboxylic acid or mixtures thereof, where the total of components a) to d) is 100% by weight, where the ratio of active ingredient to excipient mixture II) is from 1:2 to 3:1 by weight.