– CureDuchenne Funded
– CureDuchenne Cares Partner
Preclinical
Clinical
Approved
Research | Company | Drug Name | Status | |
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Renin-Angiotensin System Agonists | RASRx | MMX1902 | ![]() |
Ang(1-7)/Mas | Tarix | TXA127 | ![]() |
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Anti-fibrotic | Akashi | HT100 | ![]() |
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Anti-fibrotic | FibroGen | FG-3019; CTGF Ab | ![]() |
Research | Company | Drug Name | Status | |
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Nitric oxide donator Ca+ modulator | Myotherix | MRX-1043 | ![]() |
Anti inflammatory, Anti fibrotic | Nicox | HCT 1026 | ![]() |
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Non-hormonal steroid |
Reveragen | VBP15 | ![]() |
Ryanodine modulation | Armgo | S48168 | ![]() |
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Non Steroid Anti-inflammatory | Catabasis | Edasalonexent (previously CAT-1004) | ![]() |
NHE-1 Inhibitor | EspeRare | EMD 87 580 Rimeporide | ![]() |
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Steroid | Marathon |
EMFLAZA |
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Research | Company | Drug Name | Status | |
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Cardiac derived stem cells | Capricor | CAP-1002 | ![]() |
VIP Agonist | PhaseBio | PB1046 Vasomera | ![]() |
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Membrane sealant | Phrixus | P188 | ![]() |
Research | Company | Drug Name | Status | |
Gene editing/Stem cells | Anagenesis Biotherapies | CRISPR/Cas9 edited stem cells | ![]() |
Research | Company | Drug Name | Status | |
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Exon Skipping | BioMarin | Drisapersen | ![]() |
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Duplication Exon Skipping | K Flanigan Nationwide Ohio | rAAV.U7 snRNA exon 2 | ![]() |
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Exon Skipping | Sarepta |
Exondys51 |
![]() Accelerated Approval |
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Exon Skipping | Sarepta | SRP-4053 | ![]() |
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Exon Skipping | Sarepta | SRP-4045 | ![]() |
Exon Skipping | Synthena | Tricyclo-LNA | ![]() |
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Stereopure antisense oligonucleotide therapeutic | Wave | Exon 44 Skipping | ![]() |
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Stereopure antisense oligonucleotide therapeutic | Wave | Exon 45 Skipping | ![]() |
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Stereopure antisense oligonucleotide therapeutic | Wave | WVE-210201 (Exon 51 Skipping) | ![]() |
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Stereopure antisense oligonucleotide therapeutic | Wave | Exon 53 Skipping | ![]() |
Exon Skipping | Genethon | rAAV.U7 snRNA exon 53 | ![]() |
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Exon Skipping |
NS Pharma | Exon 53 | ![]() |
Research | Company | Drug Name | Status | |
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Genome editing |
Exonics |
CRISPR/Cas9 | ![]() |
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Gene Therapy | Bamboo |
BMB-D001 |
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Gene Therapy | Solid Bioscience |
SGT-001 |
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Gene Therapy |
Nationwide Children’s Hospital |
microdystrophin/AAV |
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Research | Company | Drug Name | Status | |
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Coenzyme Q10 analog | Santhera | Catena Idebenone | ![]() |
Mediated gene regulation |
Mitobridge |
MTB-1 |
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Research | Company | Drug Name | Status | |
Myostatin Antibody | Roche/Genentech | BMS-986089 | ![]() |
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Follistatin gene therapy | Milo Biotechnology | rAAV1-Follistatin | ![]() |
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Myostatin Antibody | Pfizer |
PF-06252616 |
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Research | Company | Drug Name | Status | |
Compensatory protein | P Martin Nationwide Ohio | GalGT2 | ![]() Preclinical |
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Utrophin upregulation | Summit | SMT C1100 | ![]() |
Compensatory protein | Tivorsan |
Biglycan |
![]() Preclinical |
Research | Company | Drug Name | Status | |
Stop Codon readthrough | Eloxx Pharma | Aminoglycoside derivatives | ![]() Preclinical |
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Nonsense mutation | PTC Therapeutics | Ataluren | ![]() Conditional Approval in EU |