Duchenne's Muscular Dystrophy (DMD)

Also known as: Duchenne's Muscular Dystrophy / Duchenne Muscular Dystrophy (DMD) / Duchenne Muscular Dystrophy / Muscular Dystrophy, Duchenne / Muscular dystrophy

DrugDrug NameDrug Description
DB05016AtalurenAtaluren is a novel, orally administered drug that targets nonsense mutations. 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. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene. This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
DB11921DeflazacortDeflazacort is a glucocorticoid used as an anti-inflammatory and immunosuppressant. It was approved in February, 2017 by the FDA for use in treatment of Duchenne muscular dystrophy (trade name Emflaza).
DB06014EteplirsenEteplirsen is a phosphoramidite morpholino sequence complementary to a portion of exon 51. [A18677] It exerts it's mechanism of action by forcing the exclusion of exon 51 from the mature DMD mRNA. [A18677]
DrugDrug NamePhaseStatusCount
DB11921Deflazacort1Completed2
DB12888Ezutromid1Completed1
DB00798Gentamicin1Completed1
DB01050Ibuprofen1Completed1
DB00883Isosorbide dinitrate1Completed1
DB00125L-Arginine1Completed2
DB05914Mesenchymal Stem Cells1Active Not Recruiting1
DB00331Metformin1Completed1
DB00203Sildenafil1Completed1
DB00820Tadalafil1Completed1
DB00675Tamoxifen1Active Not Recruiting1
DB06014Eteplirsen1 / 2Completed2
DB12645Givinostat1 / 2Completed1
DB04866Halofuginone1 / 2Terminated1
DB01277Mecasermin1 / 2Unknown Status1
DB05016Ataluren2Completed3
DB05016Ataluren2Terminated2
DB06014Eteplirsen2Active Not Recruiting1
DB06014Eteplirsen2Completed2
DB06014Eteplirsen2Recruiting1
DB12888Ezutromid2Active Not Recruiting1
DB04866Halofuginone2Terminated2
DB09081Idebenone2Completed2
DB12321Ifetroban2Not Yet Recruiting1
DB00860Prednisolone2Completed1
DB00635Prednisone2Recruiting1
DB00635Prednisone2Withdrawn1
DB00203Sildenafil2Terminated1
DB05016Ataluren2 / 3Terminated1
DB00612Bisoprolol2 / 3Recruiting1
DB12116Epigallocatechin gallate2 / 3Active Not Recruiting1
DB12645Givinostat2 / 3Recruiting1
DB00722Lisinopril2 / 3Completed1
DB09270Ubidecarenone2 / 3Completed1
DB05016Ataluren3Active Not Recruiting1
DB05016Ataluren3Enrolling by Invitation1
DB11921Deflazacort3Active Not Recruiting1
DB11921Deflazacort3Withdrawn1
DB00700Eplerenone3Completed1
DB06014Eteplirsen3Active Not Recruiting1
DB12645Givinostat3Recruiting1
DB00331Metformin3Completed1
DB04861Nebivolol3Active Not Recruiting1
DB00635Prednisone3Active Not Recruiting1
DB00635Prednisone3Completed2
DB00635Prednisone3Terminated1
DB00421Spironolactone3Completed1
DB00675Tamoxifen3Recruiting1
DB01136Carvedilol4Unknown Status2
DB00178Ramipril4Unknown Status1
DB11921DeflazacortNot AvailableApproved for Marketing1
DB00633DexmedetomidineNot AvailableCompleted1
DB13688DrisapersenNot AvailableNo Longer Available1
DB00700EplerenoneNot AvailableCompleted1
DB09081IdebenoneNot AvailableAvailable1
DB10791Influenza A virus A/california/7/2009 x-179a (H1N1) antigen (formaldehyde inactivated)Not AvailableUnknown Status1
DB01221KetamineNot AvailableCompleted1
DB00722LisinoprilNot AvailableCompleted1
DB00678LosartanNot AvailableCompleted1
DB00203SildenafilNot AvailableCompleted1
DB00820TadalafilNot AvailableCompleted1
DB00624TestosteroneNot AvailableActive Not Recruiting1