- COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
-
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.
- -
-
Paragraph 0262-0264
(2019/10/29)
-
- COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE
-
The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted bicyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.
- -
-
Page/Page column 130; 131
(2019/01/06)
-
- Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
-
SMA is an inherited disease that leads to loss of motor function and ambulation and a reduced life expectancy. We have been working to develop orally administrated, systemically distributed small molecules to increase levels of functional SMN protein. Compound 2 was the first SMN2 splicing modifier tested in clinical trials in healthy volunteers and SMA patients. It was safe and well tolerated and increased SMN protein levels up to 2-fold in patients. Nevertheless, its development was stopped as a precautionary measure because retinal toxicity was observed in cynomolgus monkeys after chronic daily oral dosing (39 weeks) at exposures in excess of those investigated in patients. Herein, we describe the discovery of 1 (risdiplam, RG7916, RO7034067) that focused on thorough pharmacology, DMPK and safety characterization and optimization. This compound is undergoing pivotal clinical trials and is a promising medicine for the treatment of patients in all ages and stages with SMA.
- Ratni, Hasane,Ebeling, Martin,Baird, John,Bendels, Stefanie,Bylund, Johan,Chen, Karen S.,Denk, Nora,Feng, Zhihua,Green, Luke,Guerard, Melanie,Jablonski, Philippe,Jacobsen, Bjoern,Khwaja, Omar,Kletzl, Heidemarie,Ko, Chien-Ping,Kustermann, Stefan,Marquet, Anne,Metzger, Friedrich,Mueller, Barbara,Naryshkin, Nikolai A.,Paushkin, Sergey V.,Pinard, Emmanuel,Poirier, Agnès,Reutlinger, Michael,Weetall, Marla,Zeller, Andreas,Zhao, Xin,Mueller, Lutz
-
p. 6501 - 6517
(2018/08/17)
-
- COMPOUNDS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS
-
The present invention provides compounds of formula (I) (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.
- -
-
Page/Page column 33
(2017/06/01)
-
- COMPOSITIONS FOR TREATING SPINAL MUSCULAR ATROPHY
-
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.
- -
-
Page/Page column 55
(2017/05/28)
-
- COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
-
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.
- -
-
Page/Page column 33
(2015/12/23)
-
- 2-ALKYL-6-CYCLOAMINO-3-(PYRIDIN-4-YL)IMIDAZO[1,2-B]-PYRIDAZINE DERIVATIVES, PREPARATION THEREOF, AND THERAPEUTIC APPLICATION THEREOF
-
The invention relates to 2-alkyl-6-cycloamino-3-(pyridin-4-yl)imidazo[1,2-b]pyridazine derivatives of the general formula (I) where: R2 is a C1-6-alkyl, C3-7-cycloalkyl, C3-7-cycloalkyl-C1-4-alkyl, C1-4-alkyloxy-C-M-alkyl, C3-7-cycloalkyloxy-C1-4-alkyl>C3-7-cycloalkyl-C1-4-alkyloxy-C1-4-alkyl, hydroxy-C1-6-alkyl, C1-4-fluoroalkyl group; R3 is a hydrogen atom or a substituent selected from halogen atoms and the C1-3 alkyl, —NR4R5, hydroxyl or C1-4 alkyloxy groups; A is a C1-7-alkylene group optionally substituted by one or two Ra groups; B is a C1-7-alkylene group optionally substituted by one or two Rb groups; L is either a nitrogen atom optionally substituted by an Rc or Rd group or a carbon atom substituted by an Re1 group and an Rd group or by two Re2 groups; Rd is a group selected from a hydrogen atom or a C1-6-alkyl, C3-7-cycloalkyl, C3-7-cycloalkyl-C1-6-alkyl, C1-6-alkylthio-C1-6-alkyl, C1-6-alkyloxy-C1-6-alkyl, C1-6-fluoroalkyl, hydroxy-C1-6-alkyl group; Rf is a C1-6-alkyl, C3-7-cycloalkyl, C3-7-cycloalkyl-C1-6-alkyl, C1-6-alkyloxy-C1-6-alkyl, C3-7-cycloalkyloxy-C1-4-alkyl, C3-7-cycloalkyl-C1-4-alkyloxy-C1-4-alkyl, hydroxy-C1-6-allyl, C1-6-fluoroalkyl or benzyl group. The invention also relates to a method for preparing same and to the therapeutic application thereof.
- -
-
Page/Page column 17
(2012/01/13)
-
- FUSED HETEROCYCLIC SULFONYLUREA COMPOUND, HERBICIDE CONTAINING THE SAME, AND METHOD OF CONTROLLING WEED WITH THE SAME
-
The present invention provides a compound represented by the formula: wherein Q represents a fused heterocyclic group, X and Y are the same or different and each represent an optionally halogenated lower alkyl group, an optionally halogenated lower alkoxy group, etc. , or a salt thereof, as well as a herbicide comprising the compound or a salt thereof, which exhibits a significant effect for control of sulfonylurea herbicide-resistant weeds in paddy fields and can reduce the number of active ingredients in a combined preparation and a method of controlling sulfonylurea herbicide-resistant weeds which comprises using the same.
- -
-
-