Abstract: A method for determining a biological response of a target (41, 42) to a soluble candidate substance includes the steps: introducing a soluble candidate substance into a laminar flow of a buffer liquid (2) to form a candidate substance solute (3) having an initial concentration profile (31); dispersing the initial concentration profile (31) to form a dispersed concentration profile (32); directing the dispersed concentration profile (32) into a detection channel (12) to form a final symmetrical concentration profile (33) therein; introducing a target into the detection channel (12) to obtain a combined concentration profile including a constant target concentration profile overlying the final symmetrical concentration profile (33); holding in the detection channel (12) at least one half of the combined concentration profile; and optically scanning the combined concentration profile to detect an optical signal representative of the biological response of the target to the soluble candidate substance.

Abstract: An apparatus (2) for the automatic testing of substances for carcinogenicity comprises: a plate support (3) on which at least one micro-well plate (1) can be placed; a movable pipetting unit (4) comprising a predetermined number of pipettes (40); a number of containers (5) containing a liquid culture medium (50), the number of containers (5) corresponding to the number of pipettes (40) of the pipetting unit (4); a dish support (6) on which a corresponding number of dishes (60) can be placed, each having a bottom and an upstanding side wall (601); a plurality of spinners (63) for spinning the dishes (60); a plurality of suction cups (62) for placing a lid (600) on each of the dishes; and a plurality of laterally arranged belts (64,65) for transporting the closed dishes (60) to an intermediate storage (8).

Abstract: A method for training staff for quality control when filling amorphous products into primary packaging. The method includes a provision step during which a test set having a plurality of primary packages filled with an amorphous product is provided, wherein at least one of the filled primary packages is afflicted by a contaminant comprising a fluorescent marker. A training step includes presenting the test set to a person to be trained, who carries out a visual quality control for detecting contaminants. The result of the training step is documented. A verification step is conducted during which the test set is irradiated by excitation light, wherein fluorescing contaminants are detected and the result of the verification step is documented. A comparison step is done during which the result of the training step and the result of the verification step are compared. Also disclosed is an inventive test kit and test set.

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.

Abstract: A method is provided for determining the efficacy of GPC3-targeting drug therapy for cancer in a patient before the start of GPC3-targeting drug therapy or for determining the continuation of GPC3-targeting drug therapy for a patient treated with GPC3-targeting therapy. The method includes determining the number of an immunocyte or an expression level of a molecule expressed on the immunocyte in a biological sample isolated from the patient before the start of GPC3-targeting drug therapy and/or the patient treated with the GPC3-targeting drug therapy, wherein when the number of the immunocyte or the expression level of the molecule expressed on the immunocyte is a predetermined value, the efficacy of the GPC3-targeting drug therapy is determined or the continuation of the GPC3-targeting drug therapy is determined. GPC3-targeting drugs and drug preparations for use according to the disclosed methods are also provided.

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

Abstract: The present invention relates to variable domains of a camelid heavy-chain antibodies directed against a phosphorylated tau protein and conjugates thereof. The present invention also relates to the use of these domains or conjugates for treating or diagnosing disorders mediated by neurofibrillary tangles, neuropil threads or dystrophic neurites, such as tauopathies.

Abstract: Provided are compositions and methods for inhibiting hypoxia-induced damage. The compositions and methods involve the use of one or more agents that can inhibit one or any combination of the genes BCL2L14, BLOC1S2, C20RF42, CPT1A, FBP1, GCNT3, RHOB, SCIN, TACR1 and TNFAIP6. Polynucleotide and non-polynucleotide agents which can be used for inhibiting one or more of the genes are included. The method involves introducing one or more gene inhibiting agents to a cell, tissue, organ, or individual such that formation of hypoxia related damage is inhibited. Kits which contain the agents and printed information about using them for inhibiting hypoxia induced damage are also included.

Abstract: The present invention is directed to a combination therapy involving a type II anti-CD20 antibody and a selective Bcl-2 inhibitor for the treatment of a patient suffering from cancer, particularly, a CD20-expressing cancer.

Abstract: The present invention relates to an oligopeptide and conjugates thereof. The present invention also relates to the use of these oligopeptide conjugates for treating or diagnosing disorders mediated by amyloid ? deposits. Finally, the present invention also relates to a coupling method for obtaining oligopeptide coupled with a substance of interest (functional conjugates).

Abstract: In the present invention a method for purifying erythropoietin comprising at least one chromatography step using a stationary phase containing hydroxyapatite is reported. The method comprises the following steps i) the erythropoietin in a solution containing Calcium-ions is brought into contact with a stationary phase containing hydroxyapatite equilibrated with a solution containing Calcium-ions and namely under conditions under which the erythropoietin binds to the stationary phase containing hydroxyapatite, ii) a solution is passed over the stationary phase containing hydroxyapatite from i) which contains less Calcium-ions than the previous solution and the erythropoietin is not detached from stationary phase containing hydroxyapatite, and iii) a further solution which contains less than 0.5 mM Calcium-ions is passed over the stationary phase containing hydroxyapatite from ii) whereby the erythropoietin is detached from the stationary phase containing hydroxyapatite.

Abstract: Compounds of Formulas Ia-d where X is S or O, mor is a morpholine group, and R3 is a monocyclic heteroaryl group, and including stereoisomers, geometric isomers, tautomers, solvates, metabolites and pharmaceutically acceptable salts thereof, are useful for modulating the activity of lipid kinases including PI3K, and for treating disorders such as cancer mediated by lipid kinases. Methods of using compounds of Formula Ia-d for in vitro, in situ, and in vivo diagnosis, prevention or treatment of such disorders in mammalian cells, or associated pathological conditions, are disclosed.

Abstract: The present invention relates to a compound having the general formula (A), optionally in the form of a pharmaceutically acceptable salt, solvate, polymorph, codrug, cocrystal, prodrug, tautomer, racemate, enantiomer, or diastereomer or mixture thereof, which are useful in treating, ameloriating or preventing a viral disease. Furthermore, specific combination therapies are disclosed.

Abstract: 5-Azaindazole compounds of Formula I, including stereoisomers, geometric isomers, tautomers, and pharmaceutically acceptable salts thereof, are useful for inhibiting Pim kinase, and for treating disorders such as cancer mediated by Pim kinase. Methods of using compounds of Formula I for in vitro, in situ, and in vivo diagnosis, prevention or treatment of such disorders in mammalian cells, or associated pathological conditions, are disclosed.

Abstract: There are provided compounds of the formula wherein: V, W, X, Y, Z, R1, R2, R3, R4, R5 and R6 are described herein. The compounds are useful for the treatment of proliferative diseases, including cancer.

Abstract: Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Abstract: The present invention relates to a compound having the general formula (V), optionally in the form of a pharmaceutically acceptable salt, solvate, polymorph, codrug, cocrystal, prodrug, tautomer, racemate, enantiomer, or diastereomer or mixture thereof, which are useful in treating, ameloriating or preventing a viral disease. Furthermore, specific combination therapies are disclosed.

Abstract: The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 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 present invention relates to a compound having the general formula (V), optionally in the form of a pharmaceutically acceptable salt, solvate, polymorph, codrug, cocrystal, prodrug, tautomer, racemate, enantiomer, or diastereomer or mixture thereof, which are useful in treating, ameloriating or preventing a viral disease. Furthermore, specific combination therapies are disclosed.

Abstract: The invention is concerned with the compounds of formula (I): and pharmaceutically acceptable salts thereof. In addition, the present invention relates to methods of manufacturing and using the compounds of formula (I) as well as pharmaceutical compositions containing such compounds. The compounds of formula (I) are LMP7 inhibitors and may be useful in treating associated inflammatory diseases and disorders such as, for example, rheumatoid arthritis, lupus and irritable bowel disease.