Research Overview

CureDuchenne FundedCureDuchenne Funded CureDuchenne Cares PartnerCureDuchenne Cares Partner
PreclinicalPreclinical ClinicalClinical ApprovedApproved DiscontinuedDiscontinued


Anti-Fibrosis Therapy

Research Company Drug Name Status
helix Renin-Angiotensin System Agonists RASRx RASRx1902 Preclinical
helixheart Renin-Angiotensin System Agonists BioPhytis BIO101 Preclinical
helix Anti-fibrotic Akashi HT100 discontinued Discontinued
heart Anti-CTGF antibody FibroGen Pamrevlumab clinical Clinical
heart HDAC inhibitor Italfarmaco Givinostat clinical Clinical


Anti-Inflammatory Therapy

Research Company Drug Name Status
helix Nitric oxide donator Ca+ modulator Myotherix MRX-1043 Preclinical
helix

Non-hormonal steroid

Reveragen Vamorolone clinical Clinical
heart Non Steroid Anti-inflammatory Catabasis Edasalonexent clinical Clinical
NHE-1 Inhibitor EspeRare Rimeporide clinical Clinical
CD49d espression inhibitor Antisense Therapeutics ATL1102 approved Clinical
Estrogen receptor inhibitor University Hospital in Basel Tamoxifen approved Clinical
heart Sterioid alternative Mallinckrodt Cosyntropin discontinued Discontinued
heart Corticosteroid PTC Therapeutics EMFLAZA approved Approved


Cardiac Function

Research Company Drug Name Status
helixheart Cardiac derived stem cells Capricor CAP-1002 clinical Clinical
Thromboxane receptor inhibitor Cumberland Ifetroban clinical Clinical
Membrane sealant Phrixus P188 clinical Clinical


Cell Therapy

Research Company Drug Name Status
Gene editing/Stem cells Anagenesis Biotherapies CRISPR/Cas9 edited stem cells Preclinical


Exon Skipping

Research Company Drug Name Status
cureduchenne cares supporter Exon Skipping BioMarin Drisapersen Exon 51 discontinued Discontinued
funded by cureduchennecureduchenne cares supporter Exon Skipping – PMO Sarepta Exondys51 Exon 51 approved Accelerated Approval
cureduchenne cares supporter Exon Skipping – PMO Sarepta Vyondys53 Exon 53 approved Accelerated Approval
cureduchenne cares supporter Exon Skipping – PMO Sarepta Casimersen Exon 45 status Clinical
cureduchenne cares supporter Exon Skipping – PMO Sarepta Exon 52 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta SRP-5051 Exon 51 status Clinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 44 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 45 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 50 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 52 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta Exon 53 status Preclinical
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave Suvodirsen Exon 51 discontinued Discontinued
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave WVE-N531 Exon 53 discontinued Discontinued
cureduchenne cares supporter Exon Skipping – PPMO NS Pharma Vitolarsen Exon 53 status Clinical
cureduchenne cares supporter Exon Skipping – PPMO NS Pharma NS-089/NCP-02 Exon 44 status Clinical
funded by cureduchennecureduchenne cares supporter Exon Skipping (AAV-ASO) Nationwide Children’s & Audentes AT702 Exon 2 duplication, Exon 1-5 status Preclinical
cureduchenne cares supporter Exon Skipping (AAV-ASO) Audentes AT751 Exon 51 status Preclinical
cureduchenne cares supporter Exon Skipping (AAV-ASO) Audentes AT753 Exon 53 status Preclinical
Exon Skipping Genethon rAAV.U7 snRNA Exon 53 status Preclinical


Gene Correction

Research Company Drug Name Status
helix Genome editing Vertex (aquired Exonics) CRISPR/Cas9 Preclinical
helixheart Gene Therapy Pfizer PF-06939926 clinical Clinical
heart Gene Therapy Sarepta SRP-9001 clinical Clinical
heart Gene Therapy Sarepta & Genethon rAAV2/8 micro-dystrophin Preclinical
heart Gene Therapy Solid Bioscience SGT-001 clinical Clinical


Mitochondrial

Research Company Drug Name Status
helixheart Coenzyme Q10 analog Santhera Idebenone clinical Clinical
heart Mitochondria biogenesis Astellas ASP0367 clinical Clinical
Mitochondria biogenesis Cardero Epicatechin clinical Clinical


Muscle Regeneration

Research Company Drug Name Status
Follistatin gene therapy Milo Biotechnology rAAV1-Follistatin discontinued Discontinued
heart Myostatin Antibody Roche RO7239361 discontinued Discontinued
heart Myostatin Antibody Pfizer PF-06252616 discontinued Discontinued


Protein Replacement

Research Company Drug Name Status
heart GALGT2 gene theray Sarepta & Nationwide Children’s Hospital GalGT2 clinical Clinical
helixheart Utrophin upregulation Summit SMT C1100 discontinued Discontinued


Stop Codon Read-Through

Research Company Drug Name Status
helixheart Nonsense mutation PTC Therapeutics Ataluren approved Conditional Approval in EU

Chart updated on 1/16/20

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