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Tofersen
go.drugbank.com/drugs/DB14782ApprovedInvestigational[A259023] However, it could potentially be due to the short timeframe of tofersen treatment, and more longterm trials are being conducted to confirm this hypothesis.[A259023] … [L46108] Although there were various causes of ALS, 2% of ALS cases are due to SOD1 mutations, with more than 200 SOD1 mutations documented.
Synonyms: -(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))R, DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-SP-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SPPonatinib
go.drugbank.com/drugs/DB08901ApprovedInvestigationalPonatinib is a novel Bcr-Abl tyrosine kinase inhibitor that is especially effective against the T315I mutation for the treatment of chronic myeloid leukemia. FDA approved on December 14, 2012.
Toremifene
go.drugbank.com/drugs/DB00539ApprovedInvestigationalToremifene is a selective estrogen receptor modulator (SERM) and a nonsteroidal antiestrogen used to treat estrogen receptor positive breast cancer.
Ursodeoxycholic acid
go.drugbank.com/drugs/DB01586ApprovedInvestigational[A256272] Due to its hydrophilicity, UDCA is less toxic than [cholic acid] or [chenodeoxycholic acid]. … [A256272,A256277] UDCA has been used to treat liver disease for decades: its first use in traditional medicine dates back more than a hundred years.
Products: ACIDO URSODESOSSICOLICO DOC GENERICI, ACIDO URSODESOSSICOLICO MYLAN GENERICSPilocarpine
go.drugbank.com/drugs/DB01085ApprovedInvestigational[A262036] Pilocarpine is used to treat dry mouth and various ophthalmic conditions, including elevated intraocular pressure and glaucoma. The usage of glaucoma by pilocarpine dates back to 1875.
Products: MINIMS PILOCARPINE NITRATE EYE DROPS 2% w/vFosphenytoin
go.drugbank.com/drugs/DB01320ApprovedInvestigationalIts main mechanism is to block frequency-dependent, use-dependent and voltage-dependent neuronal sodium channels, and therefore limit repetitive firing of action potentials.
Benzgalantamine
go.drugbank.com/drugs/DB19353ApprovedInvestigational[L51564] Benzgalantamine was approved by the FDA in July 2024 for the treatment of mild-to-moderate dementia in Alzheimer's patients.[L51534,L51564]
1D-myo-inositol 1,4,5-trisphosphate
go.drugbank.com/drugs/DB03401ExperimentalIntracellular messenger formed by the action of phospholipase C on phosphatidylinositol 4,5-bisphosphate, which is one of the phospholipids that make up the cell membrane.
ANA975
go.drugbank.com/drugs/DB06490ExperimentalANA975 is a toll-like receptor 7 agonist that is developed for the treatment of hepatitis C.
Synonyms: THIAZOLO(4,5-D)PYRIMIDIN-2(3H)-ONE, 5-AMINO-3-(2,3-DI-O-ACETYL-.BETA.-D-RIBOFURANOSYL)-Piflufolastat F 18
go.drugbank.com/drugs/DB14805ApprovedInvestigationalPiflufolastat F18, also called [F-18]-DCFPyL, is a urea-based radiopharmaceutical that binds to PSMA and allows for the visualization of cancerous prostate tissue. … It was first approved by the FDA in May 2021 under the brand name Pylarify and aims to allow for earlier and more accurate detection of suspected prostate cancer metastases or recurrences.