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Tofersen
go.drugbank.com/drugs/DB14782ApprovedInvestigational[L52735] Tofersen demonstrated efficacy in reducing the concentration of SOD1 in CSF and of neurofilament light chains in plasma over 28 weeks, although the ALS Functional Rating Scale–Revised did not
Lunsotogene parvec
go.drugbank.com/drugs/DB18278ApprovedInvestigational[L56135,L56136] This landmark decision followed designations as a Regenerative Medicine Advanced Therapy (RMAT) and Fast Track, marking the first-ever gene therapy approved to restore a neurosensory function
Givosiran
go.drugbank.com/drugs/DB15066Approved[A187991] Givosiran is the second-ever FDA-approved member of the siRNA drug class (the first being [patisiran]), a new class of drugs promising an important and exciting step forward in the treatment
Growth hormone-releasing peptide 1
go.drugbank.com/drugs/DB19259InvestigationalGrowth hormone-releasing peptide 1 is under investigation in clinical trial NCT00381602 (A Phase II, Randomized, Cross-over, Vehicle-controlled, Double-blind, Multicenter Study of the Safety, Pharmacodynamics
Ridogrel
go.drugbank.com/drugs/DB01207ExperimentalHowever, there currently are no clinical indications for preferential use of ridogrel over aspirin.
MDX-214
go.drugbank.com/drugs/DB06110ExperimentalMDX-214 consists of recombinant human epidermal growth factor (EGF) genetically linked to a fully human antibody fragment that is designed to activate cytotoxic killing of cancer cells by immune effector cells. It is developed for the tr...
IGN311
go.drugbank.com/drugs/DB04988ExperimentalIGN311 is a humanized monoclonal antibody (mab) against the Lewis Y carbohydrate antigen, a blood-group-related oligosaccharide.
Dirithromycin
go.drugbank.com/drugs/DB00954InvestigationalDirithromycin is a macrolide glycopeptide antibiotic used to treat many different types of bacterial infections, such as bronchitis, pneumonia, tonsillitis, and even skin infections.
Clonazepam
go.drugbank.com/drugs/DB01068ApprovedIllicitInvestigationalA benzodiazepine used to treat various seizures, including myotonic or atonic seizures, photosensitive epilepsy, and absence seizures, although tolerance may develop.[FDA Label] The agent has also been indicated for treating panic disord...
Osemozotan
go.drugbank.com/drugs/DB05339ExperimentalMN-305 is a novel, potent and highly selective serotonin 5-HT1A receptor agonist under development by MediciNova for the treatment of anxiety disorders beginning with Generalized Anxiety Disorder (GAD).