It’s only fair to share… Today, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older. “Today’s approval provides additional hope to the 100,000 people in the U.S., and the more than 20 million …
Category «Fast track»
Selinexor
It’s only fair to share… Selinexor セリネクソル KPT-330 UNII-31TZ62FO8F (Z)-3-[3-[3,5-bis(trifluoromethyl)phenyl]-1,2,4-triazol-1-yl]-N‘-pyrazin-2-ylprop-2-enehydrazide Formula C17H11F6N7O CAS 1393477-72-9 Mol weight 443.306 FDA, APPROVED 2019/7/3, Xpovio CAS : 1393477-72-9 (free base) 1421923-86-5 (E-isomer) 1621865-82-4 (E-isomer) Unknown (HCl) Treatment of cancer, Antineoplastic, Nuclear export inhibitor Selinexor (INN, trade name Xpovio; codenamed KPT-330) is a selective inhibitor of nuclear export used as an anti-cancer drug. It works by quasi-irreversibly binding to exportin …
FDA approves first treatment Ruzurgi (amifampridine) for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder
It’s only fair to share… FDA approves first treatment Ruzurgi (amifampridine) for children with Lambert-Eaton myasthenic syndrome, a rare autoimmune disorder The U.S. Food and Drug Administration today approved Ruzurgi (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in patients 6 to less than 17 years of age. This is the first FDA …
Beperminogene perplasmid, ベペルミノゲンペルプラスミド
It’s only fair to share… 1gctgcttcgc gatgtacggg ccagatatac gcgttgacat tgattattga 51ctagttatta atagtaatca attacggggt cattagttca tagcccatat 101atggagttcc gcgttacata acttacggta aatggcccgc ctggctgacc 151gcccaacgac ccccgcccat tgacgtcaat aatgacgtat gttcccatag 201taacgccaat agggactttc cattgacgtc aatgggtgga gtatttacgg 251taaactgccc acttggcagt acatcaagtg tatcatatgc caagtacgcc 301ccctattgac gtcaatgacg gtaaatggcc cgcctggcat tatgcccagt 351acatgacctt atgggacttt cctacttggc agtacatcta cgtattagtc 401atcgctatta ccatggtgat gcggttttgg cagtacatca atgggcgtgg 451atagcggttt gactcacggg gatttccaag tctccacccc attgacgtca …
Cavosonstat (N-91115)
It’s only fair to share… Cavosonstat (N-91115) CAS 1371587-51-7 C16H10ClNO3, 299.71 g/mol UNII-O2Z8Q22ZE4, O2Z8Q22ZE4, NCT02589236; N91115-2CF-05; SNO-6 3-chloro-4-(6-hydroxyquinolin-2-yl)benzoic acid Treatment of Chronic Obstructive Pulmonary Diseases (COPD), AND Cystic fibrosis, Nivalis Therapeutics, phase 2 The product was originated at Nivalis Therapeutics, which was acquired by Alpine Immune Sciences in 2017. In 2018, Alpine announced the sale and transfer of global rights to …
IMETELSTAT
It’s only fair to share… IMETELSTAT CAS 868169-64-6, N163L Molecular Formula, C148-H211-N68-O53-P13-S13, Molecular Weight, 4610.2379, Nucleic Acid Sequence Sequence Length: 135 a 1 c 4 g 3 tmodified DNA d(3′-amino-3′-deoxy-P-thio)(T-A-G-G-G-T-T-A-G-A-C-A-A) 5′-[O-[2-hydroxy-3-[(1-oxohexadecyl)amino]propyl] hydrogen phosphorothioate] PHASE 3, GERON, Myelodysplasia IMETELSTAT SODIUM CAS 1007380-31-5, GRN163L, GRN 163L Sodium Salt Molecular Formula: C148H198N68Na13O53P13S13 Molecular Weight: 4895.941 g/mol 5′-(O-(2-hydroxy-3-((1-oxohexadecyl)amino)propyl)phosphorothioate)-d(3′-amino-3′-deoxy-p-thio)(t-a-g-g-g-t-t-a-g-a-c-a-a), sodium salt (13) …
Icosapent ethyl, イコサペント酸エチル
It’s only fair to share… Icosapent ethyl 330.5042 , C22H34O2 cas 86227-47-6 / 73310-10-8 ethyl (5Z,8Z,11Z,14Z,17Z)-icosa-5,8,11,14,17-pentaenoate Ethyl eicosapentaenoic acid イコサペント酸エチル (5Z,8Z,11Z,14Z,17Z)-Eicosapetaenoic acid ethyl ester (all-Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl ester 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (5Z,8Z,11Z,14Z,17Z)- [ACD/Index Name] 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- 6GC8A4PAYH 86227-47-6 [RN] all-cis-5,8,11,14,17-Eicosapentaenoic Acid Ethyl Ester Timnodonic acid ethyl ester Vascepa 5,8,11,14,17-Eicosapentaenoic acid, ethyl ester, (all-Z)- (5Z,8Z,11Z,14Z,17Z)-5,8,11,14,17-Eicosapentaenoic acid ethyl …
FIRSOCOSTAT, ND 630, GS-0976, NDI-010976
It’s only fair to share… FIRSOCOSTAT, ND 630, NDI 010976, ND-630, NDI-010976 CAS: 1434635-54-7, UNII: XE10NJQ95M PHASE 2, Non-alcoholic steatohepatitis, GILEAD 1,4-dihydro-1-[(2R)-2-(2-methoxyphenyl)-2-[(tetrahydro-2H-pyran-4-yl)oxy]ethyl]-α,α,5-trimethyl-6-(2-oxazolyl)-2,4-dioxo-thieno[2,3-d]pyrimidine-3(2H)-acetic acid 2-[l-[2-(2-methoxyphenyl)-2-(oxan-4-yloxy)ethyl]-5- methyl-6-(l,3-oxazol-2-yl)-2,4-dioxo-lH,2H,3H,4H-thieno[2,3-d]pyrimidin-3-yl]-2- methylpropanoic acid 2-[1-[(2R)-2-(2-methoxyphenyl)-2-(oxan-4-yloxy)ethyl]-5-methyl-6-(1,3-oxazol-2-yl)-2,4-dioxothieno[2,3-d]pyrimidin-3-yl]-2-methylpropanoic acid CAS 1434635-54-7 Thieno[2,3-d]pyrimidine-3(2H)-acetic acid, 1,4-dihydro-1-[(2R)-2-(2-methoxyphenyl)-2-[(tetrahydro-2H-pyran-4-yl)oxy]ethyl]-α,α,5-trimethyl-6-(2-oxazolyl)-2,4-dioxo- Molecular Formula: C28H31N3O8S Molecular Weight: 569.62604 g/mol Company Nimbus Therapeutics LLC Description Small molecule allosteric inhibitor of acetyl-coenzyme A carboxylase alpha (ACACA; ACC1) and acetyl-coenzyme …
Patisiran
It’s only fair to share… Patisiran Sense strand: GUAACCAAGAGUAUUCCAUdTdT Anti-sense strand: AUGGAAUACUCUUGGUUACdTdT RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA (G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1), ALN-18328, 6024128 , ALN-TTR02 , GENZ-438027 , SAR-438037 , 50FKX8CB2Y (UNII code) for RNA, (A-U-G-G-A-A-Um-A-C-U-C-U-U-G-G-U-Um-A-C-dT-dT), complex with RNA(G-Um-A-A-Cm-Cm-A-A-G-A-G-Um-A-Um-Um-Cm-Cm-A-Um-dT-dT) (1:1) Nucleic Acid Sequence Sequence Length: 42, 21, 2112 a 7 c 7 g 4 t 12 umultistranded (2); modified CAS 1420706-45-1 Treatment of Amyloidosis, …
Vatiquinone, バチキノン
It’s only fair to share… Vatiquinone バチキノン Vatiquinone; Alpha-Tocotrienol quinone; EPI-743; UNII-6O85FK9I0X; 1213269-98-7; Vincerenone Molecular Formula: C29H44O3 Molecular Weight: 440.668 g/mol 2-[(3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trienyl]-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 2-((R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trien-1-yl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 2-[(3R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trien-1-yl]-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione 6O85FK9I0X 9604 Research Code：EPI-743; ATQ-3, BioE-743 MOA：Mitochondria Originator Edison Pharmaceuticals Developer Edison Pharmaceuticals; Sumitomo Dainippon Pharma; University of Florida; Yale University Class Alkadienes; Benzoquinones; Cyclohexenes; Small molecules Mechanism of Action Antioxidants; NQO1 modulators Orphan Drug Status Yes – Mitochondrial …