RESEARCH COMPANY COMPOUND STATUS INDICATION LOCATION CLINICAL TRIAL
Anti-fibrotic Akashi HT100 Phase 2 – Suspended as of 2/4/16 Males with Duchenne 6-20 years old Males with Duchenne 6-20 years old United States VIEW DETAILS
Anti-inflammatory Catabasis CAT-1004 Phase 2 Males with Duchenne 4-7 years old United States VIEW DETAILS
 Anti-inflammatory Steroid ReveraGen VBP15 Phase 2a Healthy males/females 18-65 years old United States VIEW DETAILS
Cardiac stem cells Capricor CAP-1002 Phase 1/2 Males/females with Duchenne 18 years and olderMales/females with Duchenne 18 years and older United States VIEW DETAILS
Coenzyme Q10 analog Santhera Catena/Raxone Idebenone Phase 3 Males/Females with Duchenne 5-16 years oldMales/Females with Duchenne 5-16 years old United States VIEW DETAILS
Exon Skipping BioMarin Drisapersen Development halted Males with Duchenne amenable to exon 51 skip United States VIEW DETAILS
BioMarin Drisapersen Development halted Males with Duchenne who previously participated in drisapersen clinical trials North America, Europe N/A
Sarepta EteplirsenExon 51 The PROMOVI study is ongoing. Exondys51 was approved 9/29/2016. Males with Duchenne having mutations amenable to exon 51 skipping United States VIEW DETAILS
Sarepta SRP-4053Exon 53 Phase 1/2 – Finished enrollment in France, Italy, & UK. Study is ongoing. EU, Males with Duchenne having genetic mutations amenable to exon 53 skipping6-15 years old, ambulatory Europe VIEW DETAILS
Sarepta SRP-4053Exon 53 Phase 3 – Currently enrolling at 14 sites in the US and 1 site in Europe with more to open soon. Males with Duchenne age 7-13, ambulatory, having mutations amenable to exon 53 skipping United States, Europe VIEW DETAILS
Sarepta SRP-4045Exon 45 Males with Duchenne age 7-13, ambulatory, having mutations amenable to exon 45 skipping VIEW DETAILS
Sarepta SRP-4045 and SRP-4053ESSENCE Males with Duchenne age 7-13, having mutations amenable to exon 45 skipping or exon 53 skipping VIEW DETAILS
Follistatin gene therapy Milo Biotech rAAVI- Follistatin Phase 1/2 Males with Duchenne Ages 7 years and older United States VIEW DETAILS
Myostatin Antibody Pfizer PF-06252616 Phase 2 Males with Duchenne 6-9 years old Canada, Europe, Japan, United States VIEW DETAILS
Regeneron REGN1033 Phase 2 TBD TBD N/A
Bristol-Meyers Squibb BMS-986089 Phase 1 Healthy males & females 18-55 years old United States VIEW DETAILS
Ryanodine modulation ARMGO/ Servier RyacalARM210/S48168 Phase 1 Duchenne Europe N/A
Steroid Marathon MP-104 Deflazacort NDA submitted 6/14/16 Males with Duchenne Ages 4-16 years old United States VIEW DETAILS
Stop Codon readthrough PTC Ataluren (PTC124) Nonsense Mutations Phase 2b Extension Study (Study 019) An Open-Label Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy Australia, Belgium, Canada, France, Germany, Israel, Italy, Spain, Sweden, United Kingdom VIEW DETAILS
PTC Ataluren (PTC124) Nonsense Mutations Phase 2b Extension study (study 016) Study of Ataluren for Previously Treated Patients With nmDBMD in the US United States VIEW DETAILS
PTC Ataluren (PTC124) Nonsense Mutations Phase 3 Confirmatory Trial (ACT DMD) Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy (ACT DMD) Australia, Canada, Europe, Korea,South America, United States VIEW DETAILS
Utrophin upregulation Summit SMT C1100 Phase 1/2 Males with Duchenne 5-13 years old Europe VIEW DETAILS

Chart updated on 4/17/17