Abstract: Novel methods for treating a condition or disorder associated with impaired neuromuscular transmission, such as MuSK myasthenia gravis (MuSK MG), in a subject are provided herein. Novel methods for treating a symptom associated with impaired neuromuscular transmission in a subject are also provided. The invention also provides multivalent binding agents for use in treating the same.

Abstract: A method of manufacturing a microstructure comprises printing a positive mold structure, filling the positive mold structure with a second material to form an elastically deformable negative mold structure, filling the negative mold structure with a third material to form the microstructure, and releasing the microstructure from the negative mold structure. Advantageously, the negative mold structure can be stretched to facilitate the release of the microstructure. For example, the microstructure comprises a chamber with capped micropillars for the generation and/or analysis of muscle tissue.

Abstract: The present invention relates to a MuSK agonist antibody, or a VH, VL or CDR3 thereof, a polynucleotide encoding said antibody or a VH, VL or CDR3 thereof or an expression vector comprising said polynucleotide or a composition comprising said antibody, polynucleotide or expression vector and their use in methods of preventing and treating diseases and conditions associated with an impaired neuromuscular transmission.

Abstract: Novel methods for treating condition, disorder and/or symptom which is alleviated by the inhibition of neuromuscular transmission in a subject are provided herein. The invention also provides binding agents for use in treating the same.

Abstract: The invention is related to facioscapulohumeral muscular dystrophy (FSHD) and in particular therapeutic means and methods for the treatment of the disease. In one aspect the invention provides a composition for use in the treatment of a subject that has FSHD which composition has a therapeutically effective amount of a substance which increases the level and/or activity of a protein encoded by a structural maintenance of chromosomes flexible hinge domain containing 1 (SMCHD1) gene in cells of the subject. The invention also provides means and methods for typing FSHD.

Abstract: The disclosure provides methods and compositions for inducing exon-skipping in a dysferlin pre-mRNA useful, e.g., in restoring function in a dysferlin deficiency. The disclosure also provides improved methods and compositions for generally inducing exon-skipping in a pre-mRNA.

Abstract: The present invention provides heavy chain variable domain antibodies (VHH) for preventing and/or dissolving aggregates. VHH of the invention are preferably used in the treatment of human diseases that are associated with the formation of aggregates in the body. The invention further provides, among others, means and methods for selecting and using VHH.