Duchenne Muscular Dystrophy

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Retrieved

2022-04-26

Source

Gard

Trials

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Genes

DMD, LTBP4, TGFB1, CD4, POSTN, CCL2, CTSS, LINC02694, ADCY8, SPP1, DOCK1, XYLT1, SHKBP1, RGS6, DAG1, ADCY1, UTRN, OTC, MSTN, PTPN1, GK, ACE, BEST1, MB, TTN, MMP9, MMP2, SSPN, CCN2, ADIPOQ

DMD, LTBP4, TGFB1, CD4, POSTN, CCL2, CTSS, LINC02694, ADCY8, SPP1, DOCK1, XYLT1, SHKBP1, RGS6, DAG1, ADCY1, UTRN, OTC, MSTN, PTPN1, GK, ACE, BEST1, MB, TTN, MMP9, MMP2, SSPN, CCN2, ADIPOQ, TNF, MIR206, GH1, LAMA2, NOS1, PLF, NR3C2, NR0B1, MIR21, TNFRSF11B, G6PD, ACTB, PROM1, DCN, SGCA, TIMP1, SMN1, SLN, GJA1, AAVS1, FSD1L, PPARD, FSD1, FN1, ELP1, AR, NEB, IL6, BGN, HPX, AQP4, BMI1, IL1B, CD38, PART1, FST, CAPN2, P2RX7, MFAP1, NFE2L2, GABPA, DYSF, IGF1, NOS2, KL, HLA-B, PIK3C2A, PDGFRA, TPPP3, HTC2, ST8SIA4, NR3C1, LGALS1, SLMAP, PLA1A, HLA-A, VEGFA, SMN2, PLAU, MIR31, RELA, CAV3, CAPN3, CAPN1, CA3, BTF3P11, BRCA1, BMP4, MIR29C, SYT1, CLK1, APRT, APC, ANGPT1, MIR483, SRY, SGCG, CCR2, LINC01672, H3P14, CFTR, DTNBP1, TLR4, FAM168B, CMYA5, GORASP1, WNK1, TM7SF2, NLRP3, CYBB, TUBB6, TIMP2, CSF3, PDE5A, TIMP3, ST14, TGM2, TNFRSF11A, TIMP4, HAP1, LARGE1, IL1RL1, TNNI3, TNFSF11, TRBV20OR9-2, DENR, AIMP2, TGFBR2, TGFA, THAS, VIM, TERT, VCAM1, DYNLT3, WNT7A, CCDC6, THBS1, THBS4, VCP, CMAHP, VCL, TERF1, SERPINA3, NOG, PANX3, RAB40AL, RMDN2, HJV, MAGEB16, RBM45, B4GALNT2, PRRT2, CPP, ATAD1, TNFAIP8L2, MMEL1, SCAF1, RAB40C, RNF213, H19, ACTBL2, CCL28, TRIM72, COMMD3-BMI1, MUPP, MIR675, POTEM, MIR486-1, MIR499A, POTEKP, FAM111B, MIR30C2, MIR30C1, MIR200C, MIR150, MIR146A, MIRLET7C, NLN, ASH1L, LIPG, ADAMTS5, SYNE1, SYNM, KDM6B, TRIM32, ACOT7, CORO1A, AHSA1, PANX1, CARM1, PPIF, HDAC9, SDC3, FHL5, GRAP2, SUN1, SUN2, SYBU, EFEMP2, RMDN3, SNTG1, PTRH2, TRPV2, TLR7, RMDN1, KLF15, PAMR1, SGSM3, SOX9, HPGDS, FGF21, POLDIP2, RNF19A, EIF3K, PITPNA, SNTA1, FKTN, FABP5, F9, F8, EZH2, ETFA, ERG, ERBB2, ENO1, EMD, ELAVL2, E2F1, DTNB, DPP4, DPEP1, DNAH8, ACSL4, FHL1, DHFR, GDNF, HSPG2, DNAJB2, HPRT1, HP, HMOX1, HLA-E, HLA-DRB1, HLA-DQA1, HLA-C, HIVEP1, HIF1A, GPX3, GPC1, GLP1R, GLB1, SEPTIN1, DFNX3, SHOX, CEACAM1, BCL2, ATP2A2, AQP1, APP, BIRC2, AGT, JAG1, AGER, ADRB2, PARP1, ADM, ACVR2B, ACTN3, ACTG2, ACTG1, BGLAP, BRCA2, CTSL, CAPNS1, CTSB, VCAN, MAPK14, CRK, CNR1, CCR5, CHI3L1, CTSC, CDKN3, CDKN1C, CD68, CD40, CASQ1, CASP4, CASP3, TNC, IFNA1, IFNA13, PRKAA1, PPID, PMP22, PLXNB1, PLN, PKD1, PITX2, PDE4A, PAX7, PAX3, SERPINE1, PAH, P2RX4, NPC1, NOTCH1, NGF, PRG2, PRKAA2, IFNG, PRKAB1, SGCD, SELENOP, CXCL11, S100B, RYR1, RGS12, RET, MOK, PTH, PTCH1, TAS2R38, MAPK1, PRKCQ, PRKCB, PRKCA, NFKB1, NF2, NCAM1, MYOD1, LRPAP1, LGALS3, LEP, KDR, ITPR3, ITPR1, ITGA7, ITGA5, ITGA4, ITGA6, INSR, IDO1, IL17A, IL10, IGF2R, MAFD2, MAOA, MAOB, MMP10, MYD88, MYCL, MYC, MUC1, MS, MRC1, MMP7, MAS1, MMP1, MIF, KITLG, MAP3K5, CD46, MBL2, H3P47

Drugs

2'-O-methyl-phosphorothioate oligonucleotide, 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine, 20- hydroxyecdysone

2'-O-methyl-phosphorothioate oligonucleotide, 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine, 20- hydroxyecdysone, 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole, Adeno-associated viral vector containing a modified U7-snRNA gene, Adeno-associated viral vector containing modified U1 snRNA, Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene, Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene, Angiotensin (1-7), Ataluren ( TRANSLARNA ), Cosyntropin, Eteplirsen ( EXONDYS 51 ), Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast, Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor, Ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast, Exon 44 specific phosphorothioate oligonucleotide, Exon 45 specific phosphorothioate oligonucleotide, Exon 51 specific phosphorothioate oligonucleotide, Exon 52 specific phosphorothioate oligonucleotide, Exon 53 specific phosphorothioate oligonucleotide, Exon 55 specific phosphorothioate oligonucleotide, Givinostat, Golodirsen ( Vyondys 53 ), Halofuginone hydrobromide, Humanised monoclonal antibody against myostatin, Metformin and L-citrulline, N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide, Naproxcinod, Pamrevlumab, R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride, RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl] N,N-dimethylaminophosphonamidate, RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[------], octahydrochloride, Rasagiline, Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter, Recombinant fusion protein consisting of the extracellular portion of human activin receptor IIB linked to the human IgG1 Fc domain, Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA, Tamoxifen citrate, Vamorolone, Viltolarsen, deflazacort ( Emflaza ), idebenone ( SAN IDEBENONE, SOVRIMA, RAXONE ), rimeporide

Interested in hearing about new therapies?

Duchenne muscular dystrophy (DMD) affects the muscles, leading to muscle wasting that gets worse over time. DMD occurs primarily in males, though in rare cases may affect females. The symptoms of DMD include progressive weakness and loss (atrophy) of both skeletal and heart muscle. Early signs may include delayed ability to sit, stand, or walk and difficulties learning to speak. Muscle weakness is usually noticeable in early childhood. Most children with DMD use a wheelchair by their early teens. Heart and breathing problems also begin in the teen years and lead to serious, life threatening complications. DMD is caused by genetic changes (DNA variants) in the DMD gene. DMD is inherited in an X-linked recessive pattern and may occur in people who do not have a family history of DMD. Diagnosis of DMD is based on the symptoms, clinical exam, and the result of a biopsy to remove a small piece of muscle for examination under a microscope. The result of genetic testing may also help confirm the diagnosis. While there is no known cure for DMD, there are treatments that can help control symptoms.

Becker muscular dystrophy (BMD), a milder form of muscular dystrophy, is also caused by DNA variants in the DMD gene.