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Displaying results 1441 - 1450 of 10000 in total
  1. Drug

    Zilganersen

    go.drugbank.com/drugs/DB18161
    ApprovedInvestigational

    [L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons … [L64096] The FDA approved zilganersen on September 3, 2026 for the treatment of Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment for the condition

  2. Drug

    Delandistrogene moxeparvovec

    go.drugbank.com/drugs/DB16802
    ApprovedInvestigational

    [L47026] DMD is an X-linked genetic disorder characterized by mutations in the dystrophin gene, leading to a deficiency in functional dystrophin protein. … [A260256] DMD tends to be more prevalent in males.

  3. Drug

    Eflornithine

    go.drugbank.com/drugs/DB06243
    ApprovedInvestigationalWithdrawn

    [A262823,L49318] Subsequently, on December 14, 2023, the FDA approved eflornithine again but under the brand name IWILFIN as an oral maintenance therapy to reduce the risk of relapse in adult and pediatric … a 68% reduction in the risk of death were observed.

  4. Drug

    Sonidegib

    go.drugbank.com/drugs/DB09143
    ApprovedInvestigational

    Sonidegib is a Hedgehog signaling pathway inhibitor (via smoothened antagonism) developed as an anticancer agent by Novartis. It was FDA approved in 2015 for the treatment of basal cell carcinoma.

    Synonyms: N-[6-(cis-2,6-dimethylmorpholin-4-yl)pyridin-3-yl]-2-methyl-4'-(trifluoromethoxy)[1,1'-biphenyl]-3-carboxamide
  5. Drug

    Ferric cation

    go.drugbank.com/drugs/DB13949
    ApprovedWithdrawn

    Iron is an essential element involved in various metabolic processes, including oxygen transport, deoxyribonucleic acid (DNA) synthesis, and energy production in electron transport [A32514]. … By mass, it is the most common element on Earth.

  6. Drug

    Risperidone

    go.drugbank.com/drugs/DB00734
    ApprovedInvestigational

    [L12885] Schizophrenia and various mood disorders are thought to be caused by an excess of dopaminergic D2 and serotonergic 5-HT2A activity, resulting in overactivity of central mesolimbic pathways … Risperidone is thought to reduce this overactivity through inhibition of dopaminergic D2 receptors and serotonergic 5-HT2A receptors in the brain.

  7. Drug

    Moxisylyte

    go.drugbank.com/drugs/DB09205
    Approved

    Moxisylyte, denominated as thymoxamine in the UK, is a specific and orally active α1-adrenergic antagonist. … [L1172] This drug was developed by the Japanese company Fujirebio and also by the American company Iolab in the late 80s.

    Categories: Drugs Used in Erectile Dysfunction
  8. Drug

    Etranacogene dezaparvovec

    go.drugbank.com/drugs/DB16791
    ApprovedInvestigational

    Hemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX. … [L44156] It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.

  9. Drug

    Inebilizumab

    go.drugbank.com/drugs/DB12530
    ApprovedInvestigational

    [L52705] In December 2025, the FDA approved an additional indication for generalized myasthenia gravis. [L54838] … [A214280, A214295] On January 16, 2024, inebilizumab also received Health Canada approval for the same indication.

  10. Drug

    Asciminib

    go.drugbank.com/drugs/DB12597
    ApprovedInvestigational

    [A241065] Asciminib is unique in that it acts as an allosteric inhibitor, binding at the myristoyl pocket of the BCR-ABL1 protein and locking it into an inactive conformation. … More specifically, it is an inhibitor of the ABL1 kinase activity of the BCR-ABL1 fusion protein[L38995] which serves as a driver of CML proliferation in most patients with the disease.