Abstract: Described herein are compounds that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.

Abstract: Described herein are compounds that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.

Abstract: Described herein are compounds that modulate splicing of a pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.

Abstract: The invention provides 7-phenoxy-N-(3-azabicyclo[3.2.1]octan-8-yl)-6,7-dihydro-5H-pyrrolo[1,2-b][1,2,4]triazol-2-amine derivatives and related compounds of the general formula (I) wherein R1, Ar, n and m are as described herein, compositions including the compounds, processes of manufacturing the compounds and the compounds for use in methods of medical treatment. The present compounds are as gamma-secretase modulators for the treatment of diseases associated with the deposition of ?-amyloid in the brain, such as Alzheimer's disease, cerebral amyloid angiopathy, cochlear synaptopathy, hearing loss, hereditary cerebral hemorrhage with amyloidosis-Dutch type (HCHWA-D), multi-infarct dementia, dementia pugilistica and Down syndrome. The present description discloses the preparation of exemplary compounds as well as pharmacological data thereof (e.g. pages 54 to 74; examples 1 to 64; table). An exemplary compound is e.g. (R)-7-(3,5-difluorophenoxy)-N-((1R,5S,8s)-3-(6-methoxypyridazin-4-yl)-3-azabicyclo[3.2.

Abstract: The present invention provides compounds of formula I or II: wherein X1, X3, R1, R2, R3, R4 and R5 are as described herein, as well as pharmaceutically acceptable salts thereof. 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 invention provides new bicyclic heteroaryl compounds having the general formula (I) wherein R1, Ar, n and m are as described herein, compositions including the compounds, processes of manufacturing the compounds and methods of using the compounds.

Abstract: The invention provides novel heterocyclic compounds having the general formula (I), and pharmaceutically acceptable salts thereof, wherein the variables are as described herein. Further provided are pharmaceutical compositions including the compounds, processes of manufacturing the compounds and methods of using the compounds as medicaments, in particular methods of using the compounds for the treatment or prevention of acute neurological disorders, chronic neurological disorders and/or cognitive disorders.

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. 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: Provided herein are compounds of Formula (I): and forms thereof, including compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract: The invention relates to a compound of formula (I) wherein R1—R4 and A1-A3 are as defined in the description and in the claims. The compound of formula (I) can be used as a medicament.

Abstract: The invention relates to a compound of formula (I) wherein R1-R4 and A1 are as defined in the description and in the claims. The compound of formula (I) can be used as a medicament.

Abstract: The invention relates to a compound of formula (I) wherein X, R1-R4 are as defined in the description and in the claims. The compound of formula (I) can be used as a medicament.

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. 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: Provided herein are compounds of Formula (I): and forms thereof, wherein w1, w2, w3, w4, w5, w6 and w7 are as defined herein, including compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract: The present invention provides compounds of formula I or II: wherein X1, X3, R1, R2, R3, R4 and R5 are as described herein, as well as pharmaceutically acceptable salts thereof. 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 invention provides novel compounds having the general formula (I) wherein R1, R2, R3, R4 and R5 are as defined herein, compositions including the compounds and methods of using the compounds.

Abstract: The invention provides novel compounds having the general formula (I) wherein R1, R2, R3 and n are as defined herein, compositions including methods of making the compounds and using the compounds as ATX inhibitors.

Abstract: The invention provides new heterocyclic compounds having the general formula (I) wherein A, B, L, Y1 to Y4 and R1 to R6 are as described herein.

Abstract: The invention provides novel compounds having the general formula I wherein R1, R2, and R3 are as defined herein, compositions including the compounds and methods of using the compounds.

Abstract: The present invention relates to a process for synthesizing large scale a compound of formula (I), or pharmacuetically acceptable salt thereof, which is useful as the key intermediate for the synthesis of compounds for prophylaxis and treatment of a disease associated with the deposition of ?-amyloid in the brain, in particular Alzheimer's disease, and other diseases such as cerebral amyloid angiopathy, hereditary cerebral hemorrhage with amyloidosis, Dutch-type (HCHWA-D), multi-infarct dementia, dementia pugilistica and Down syndrome.