– CureDuchenne Funded   – CureDuchenne Cares Partner

preclinical Preclinical clinical Clinical approved Approved discontinued Discontinued
Research Company Drug Name Status
Renin-Angiotensin System Agonists RASRx RASRx1902 Preclinical
Ang(1-7)/Mas Tarix TXA127 Preclinical
Anti-fibrotic Akashi HT100 discontinuedDiscontinued
Anti-fibrotic FibroGen Pamrevlumab 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 Vamorolone Clinical
Ryanodine modulation Armgo S48168 Clinical
Non Steroid Anti-inflammatory Catabasis Edasalonexent Clinical
NHE-1 Inhibitor EspeRare Rimeporide Clinical
Corticosteroid PTC Therapeutics

EMFLAZA

approvedApproved
Research Company Drug Name  Status
Cardiac derived stem cells Capricor CAP-1002 Clinical
VIP Agonist PhaseBio PB1046 Vasomera  Preclinical
Membrane sealant Phrixus P188  Clinical
Research Company Drug Name  Status
Gene editing/Stem cells Anagenesis Biotherapies CRISPR/Cas9 edited stem cells  Preclinical
Research Company Drug Name Status
cureduchenne cares supporter Exon Skipping BioMarin Drisapersen
Exon 51
discontinuedDiscontinued
funded by cureduchenne Duplication Exon Skipping Nationwide
Children’s
rAAV.U7 snRNA
Exon 2
status Preclinical
funded by cureduchennecureduchenne cares supporter Exon Skipping – PMO Sarepta Exondys51
Exon 51
approvedAccelerated Approval
cureduchenne cares supporter Exon Skipping – PMO Sarepta Golodirsen
Exon 53
statusClinical
cureduchenne cares supporter Exon Skipping – PMO Sarepta Casimersen
Exon 45
statusClinical
cureduchenne cares supporter Exon Skipping – PMO Sarepta Exon 52 status Preclinical
cureduchenne cares supporter Exon Skipping – PPMO Sarepta SRP-5051
Exon 51
statusClinical
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
Exon Skipping Synthena Tricyclo-LNA status Preclinical
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave Suvodirsen
Exon 51
statusClinical
cureduchenne cares supporter Stereopure antisense oligonucleotide Wave Exon 53 status Preclinical
Exon Skipping Genethon rAAV.U7 snRNA
Exon 53
status Preclinical
Exon Skipping NS Pharma Exon 53 statusClinical
Research Company Drug Name  Status
Genome editing Exonics CRISPR/Cas9  Preclinical
Gene Therapy Pfizer PF-06939926 Clinical
Gene Therapy Sarepta SRP-9001 Clinical
Gene Therapy Sarepta rAAV2/8
micro-dystrophin
 Preclinical
Gene Therapy Solid Bioscience SGT-001 Clinical
Research Company Drug Name  Status
Coenzyme Q10 analog Santhera Idebenone Clinical

Mediated gene regulation

Mitobridge

MA-0211

 Clinical
Research Company Drug Name  Status
Myostatin Antibody Roche RO7239361 Clinical
Follistatin gene therapy Milo Biotechnology rAAV1-Follistatin Clinical
Myostatin Antibody Pfizer

PF-06252616

discontinuedDiscontinued
Research Company Drug Name  Status
Compensatory protein  Nationwide Children’s Hospital GalGT2 Clinical
Utrophin upregulation Summit SMT C1100 discontinuedDiscontinued
Compensatory protein Tivorsan

Biglycan

Preclinical
Research Company Drug Name  Status
Stop Codon read-through Eloxx Pharma ELX-02 Preclinical
Nonsense mutation PTC Therapeutics Ataluren approvedConditional Approval in EU