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AAV9/MFSD8
go.drugbank.com/drugs/DB18616ExperimentalAAV9/MFSD8 is a gene therapy developed by Neurogene Inc.
Congenital aplastic anaemia
go.drugbank.com/conditions/DBCOND0007164Drugs: OxymetholoneSynonyms: Constitutional aplastic anemia, NOS, Constitutional aplastic anemia NOS (disorder)Cystic Fibrosis (CF)
go.drugbank.com/conditions/DBCOND0040212Synonyms: Cystic fibrosis NOS, Cystic fibrosis NOS (disorder)Fat malabsorption
go.drugbank.com/conditions/DBCOND0020593Drugs: PancrelipaseSynonyms: Steatorrhea, NOS, Steatorrhoea, NOSMyoclonic seizures
go.drugbank.com/conditions/DBCOND0022716Synonyms: Myoclonic seizure NOS, (Myoclonus NOS) or (Unverricht-Lundborg disease) or (myoclonic seizure)CX501
go.drugbank.com/drugs/DB05174InvestigationalIt is developed by Cellerix and is in phase I of clinical trials.
BGB-DXP593
go.drugbank.com/drugs/DB16357Investigationalthe clinical trial NCT04551898 (Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) Neutralizing Antibody BGB-DXP593 in Participants With Mild-to-Moderate Coronavirus Disease 2019 (COVID-19)) by
Devafidugene civaparvovec
go.drugbank.com/drugs/DB16790InvestigationalDeveloped by Sangamo Therapeutics, Inc., devafidugene civaparvovec is being investigated for the treatment of mucopolysaccharidosis Type I (MPS I).[L46716]
SCIB1
go.drugbank.com/drugs/DB18439InvestigationalSCIB1 is an investigational cancer vaccine being developed by Scancell Ltd. It is being investigated for the treatment of melanoma.
NGN-401
go.drugbank.com/drugs/DB18594InvestigationalDeveloped by Neurogene Inc., it is being investigated for the treatment of Rett syndrome.