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Casimersen
go.drugbank.com/drugs/DB14984ApprovedInvestigational[A218186] Casimersen is the most recent in a line of approved PMOs for treating DMD, including [eteplirsen] and [viltolarsen]. … [A218171, A218176, A229988] The application of antisense oligonucleotides in DMD patients with specific mutations allows for exon skipping to produce truncated BMD-like dystrophin proteins, which restore
Categories: Compounds used in a research, industrial, or household settingSomatrem
go.drugbank.com/drugs/DB09098ApprovedWithdrawncontinues to be mired in persistent bioethical debate [A32292]. … Despite the ability of almost all contemporary recombinant growth hormones to cause definite and demonstrable increases in growth rate in patients who are administered the drug, the use of these agents
Mixtures: Protropin Inj Pws 10mg/vial, Protropin - Kit Im Sc 10mg/vialPalbociclib
go.drugbank.com/drugs/DB09073ApprovedInvestigational[L5867] It was originally FDA approved on March 2015 for the treatment of HR-positive, HER2-negative advanced or metastatic breast cancer and its indications were updated in April 2019 to include male … Palbociclib is a piperazine pyridopyrimidine[A176792] that acts in the cell cycle machinery.
Voxelotor
go.drugbank.com/drugs/DB14975Approved[T734] Voxelotor was granted accelerated FDA approval on November 25 2019, as it is likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition … The EMA approved the use of voxelotor for the treatment of hemolytic anemia associated with sickle cell disease in February 2022.[L41419,L41424]
Agomelatine
go.drugbank.com/drugs/DB06594ApprovedInvestigationalIn 2006 Servier sold the rights to develop Agomelatine in the US to Novartis. The development for the US market was discontinued in October 2011. … The Committee for Medical Products for Human Use (CHMP) recommended refusal of marketing authorization on 27 July 2006. The major concern was that efficacy had not been sufficiently shown.
Ataluren
go.drugbank.com/drugs/DB05016ApprovedWithdrawnThis drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications. … Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk.
Ibalizumab
go.drugbank.com/drugs/DB12698ApprovedInvestigationalThis drug was approved in March 2018 for the management of treatment-resistant HIV [L1554]. … It is an advanced and current antibody in development for the treatment of HIV/AIDS. It has been developed by Taimed biologics and Theratechnologies [L1558, L1554].
Categories: Antivirals for Systemic Use, Antiinfectives for Systemic UsePropoxycaine
go.drugbank.com/drugs/DB09342ApprovedUsed in combination with procaine, it was the only dental local anesthetic available in cartridge form [L1592]. … Although no longer available in the United States, this medication was used in combination with procaine to aid in anesthesia during dental procedures [L1592].
Dabigatran etexilate
go.drugbank.com/drugs/DB06695ApprovedInvestigational[A177463] In contrast to warfarin, because its anticoagulant effects are predictable, lab monitoring is not necessary.[A177463] Dabigatran etexilate was approved by the FDA in 2010.[L6022] … [A177463, A6970, L34675, L34680] Dabigatran etexilate may be used to decrease the risk of venous thromboembolic events in patients in whom anticoagulation therapy is indicated.
Praziquantel
go.drugbank.com/drugs/DB01058ApprovedInvestigationalVet approved[A263201] The efficacy of praziquantel in treating parasitic flatworms infection with low cost (~US$0.20 drug cost to treat a child) makes it an integral to WHO's plan to eliminate schistosomiasis by 2030 … [A263206,A263211] Despite being approved since 1980, the exact mechanism of action is yet to be elucidated.[A263211]
Mixtures: Eqvalan Duo Oral Paste for Horse