Abstract: The invention relates to 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one, also known as risdiplam, for use in the treatment of spinal muscular atrophy (SMA) with GYM329, its pharmaceutical composition to be used in the treatment of SMA, its methods of treatment thereof.

Abstract: The present invention provides pharmaceutical compositions comprising a compound of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the pharmaceutical compositions comprising a compound of formula (I) and their use as medicaments.

Abstract: The present invention relates to compositions of recombinant polynucleic acid or RNA constructs comprising a first RNA sequence, a second RNA sequence, and a linker RNA sequence, wherein the linker RNA links the first RNA sequence and the second RNA sequence. In addition, linkers that enhance the cleavage of recombinant RNA constructs are described. Also disclosed herein is the use of the compositions in treating diseases and in modulating expression of two or more genes.

Abstract: The present invention relates to compositions and methods for modulating expression of genes, comprising recombinant polynucleic acid or RNA constructs comprising a first RNA sequence encoding a gene of interest, and a second RNA sequence comprising at least two genetic elements that modulate expression of one or more target RNAs. The recombinant RNA constructs described herein induce an immune response in a human cell that is lower than the immune response induced by a corresponding recombinant RNA construct comprising the first RNA sequence encoding a gene of interest and a corresponding second RNA sequence comprising at most one of the at least two genetic elements. Also disclosed herein is the use of the compositions in treating diseases and in modulating expression of two or more genes.

Abstract: The present invention relates to a mRNA and a therapeutic composition thereof for use in a method of treating lower urinary tract symptoms.

Abstract: The present invention provides pharmaceutical compositions comprising a compound of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the pharmaceutical compositions comprising a compound of formula (I) and their use as medicaments.

Abstract: The present invention relates to compositions of recombinant polynucleic acid constructs comprising at least one nucleic acid sequence encoding an siRNA capable of binding to a target mRNA and at least one nucleic acid sequence encoding a gene of interest. Also disclosed herein is use of the compositions in treating cancers and in simultaneously modulating expression of two or more genes.

Abstract: The present invention relates to compositions of recombinant polynucleic acid constructs comprising at least one nucleic acid sequence encoding an siRNA capable of binding to a target mRNA and at least one nucleic acid sequence encoding a gene of interest. Also disclosed herein is use of the compositions in treating a disease or a condition and in simultaneously modulating expression of two or more genes.

Abstract: The present invention relates to a mRNA comprising a nucleic acid sequence encoding a protein and a signal peptide and a transcription unit, an expression vector or a gene therapy vector comprising a nucleic acid encoding a protein and a signal peptide. Also disclosed herein is a therapeutic composition comprising said mRNA, transcription unit, expression vector or gene therapy vector and use of the therapeutic composition in treating a disease or a condition.

Abstract: The present invention relates to a FoxM1 gene splicing modifier selected from a compound of formula (I) or of formula (VI) wherein A and B are as defined herein, or a pharmaceutically acceptable salt thereof, for use in the treatment, prevention and/or delay of progression of cancer.

Abstract: The present invention provides pharmaceutical compositions comprising a compound of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the pharmaceutical compositions comprising a compound of formula (I) and their use as medicaments.

Abstract: The invention discloses a screening method for the identification of new compounds for use in the treatment of cancer.

Abstract: The invention discloses a screening method for the identification of new compounds for use in the treatment of cancer.

Abstract: The present invention provides pharmaceutical compositions comprising a compound of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the pharmaceutical compositions comprising a compound of formula (I) and their use as medicaments.

Abstract: The present invention provides compounds of formula (I) wherein A, R1, R2 and R3 are as described herein, as well as pharmaceutically acceptable salts thereof for use in the treatment, prevention and/or delay of progression of amyotrophic lateral sclerosis (ALS). Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.

Abstract: The present invention relates to a FoxM1 gene splicing modifier selected from a compound of formula (I) or of formula (VI) wherein A and B are as defined herein, or a pharmaceutically acceptable salt thereof, for use in the treatment, prevention and/or delay of progression of cancer.

Abstract: The invention discloses a screening method for the identification of new compounds for use in the treatment of cancer.

Abstract: The invention discloses a screening method for the identification of new compounds for use in the treatment of cancer.

Abstract: The invention discloses a screening method for the identification of new compounds for use in the treatment of cancer.

Abstract: A conjugate consisting of an insulin-like growth factor-1 (IGF-I) variant and one or two poly(ethylene glycol) group(s), characterized in that said IGF-I variant has an amino acid alteration at up to three amino acid positions 27, 37, 65, 68 of the wild-type IGF-I amino acid sequence so that one or two of said amino acids is/are lysine and amino acid 27 is a polar amino acid but not lysine, is conjugated via the primary amino group(s) of said lysine(s) and said poly(ethylene glycol) group(s) have an overall molecular weight of from 20 to 100 kDa is disclosed. This conjugate is useful for the treatment of neurodegenerative disorders like Alzheimer's Disease.