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Eteplirsen
go.drugbank.com/drugs/DB06014ApprovedInvestigationalEteplirsen was the first treatment for DMD approved by the FDA.[A244925] … [L40015] Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle deterioration and premature death most commonly due to respiratory or cardiac complications.
Miglustat
go.drugbank.com/drugs/DB00419ApprovedInvestigationalMiglustat was first developed as an anti-HIV agent in the 1990s. … It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids.
Synonyms: 1,5-(butylimino)-1,5-dideoxy, D-glucitolTofersen
go.drugbank.com/drugs/DB14782ApprovedInvestigationalTofersen is under an intrathecally administered antisense oligonucleotide targeting the mutated SOD1 gene that causes amyotrophic lateral sclerosis (ALS). … [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: 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-SPLacidipine
go.drugbank.com/drugs/DB09236ApprovedIt is not currently FDA-approved. … Due to its long duration of action, lacidipine does not lead to reflex tachycardia [A31536].
Products: LACIDIPINA DOC GENERICIFollitropin
go.drugbank.com/drugs/DB00066ApprovedInvestigationalHowever, these small differences do not affect the bioactivity compared to natural FSH. … Follitropin may also be used to cause the ovary to produce several follicles, which can then be harvested for use in gamete intrafallopian transfer (GIFT) or in vitro fertilization (IVF).
Mixtures: PERGOVERIS 150 IU/75 IU ENJEKSİYONLUK ÇÖZELTİ İÇİN TOZ VE ÇÖZÜCÜ, 1 ADET, PERGOVERIS 150 IU/75 IU ENJEKSİYONLUK ÇÖZELTİ İÇİN TOZ VE ÇÖZÜCÜ, 3 ADETProducts: Puregon (600 Iu), GONAL - F 75 IUMethadone
go.drugbank.com/drugs/DB00333ApprovedInvestigational[A185888,A185891,A185897] Compared with [morphine], the gold standard reference opioid, methadone also acts as an agonist of κ- and σ-opioid receptors, as an antagonist of the N-methyl-D-aspartate (NMDA … Methadone is a potent synthetic analgesic that works as a full µ-opioid receptor (MOR) agonist and N-methyl-d-aspartate (NMDA) receptor antagonist.
Synonyms: dl-MethadoneProducts: Metadol-DVaricella zoster vaccine (recombinant)
go.drugbank.com/drugs/DB13924ApprovedInvestigationalhave shown greater than 90% efficacy in adults aged 50 and older, with 89% efficacy in preventing postherpetic neuralgia in patients 70 years and older and 91% efficacy in patients 50-70 years of age [FDA … PHN can last for days, months, or even years following resolution of shingles.
Iptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigational[A262581] On December 6th, 2023, Iptacopan under the brand name Fabhalta was approved by the FDA for the treatment of adults with PNH. … Due to this mutation, all progeny erythrocytes will lack the glycosyl phosphatidylinositol–anchored proteins that normally anchor 2 membrane proteins, CD55 and CD59, that protect blood cells against the
Zenocutuzumab
go.drugbank.com/drugs/DB15559ApprovedInvestigational[L56179] This approval was expedited following receipt of an FDA Commissioner’s National Priority Voucher. … [L51983] In May 2026, zenocutuzumab was granted additional approval by the US FDA for use in NRG1-positive cholangiocarcinoma.
Pralatrexate
go.drugbank.com/drugs/DB06813ApprovedInvestigational[A246703] Pralatrexate was approved by the FDA on September 24, 2009. … Pralatrexate is an antifolate for the treatment of relapsed or refractory peripheral T-cell lymphoma [L37674].