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