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Research Overview

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Preclinical

Clinical

Approved

Discontinued

Anti-Fibrosis Therapy

	Research	Company	Drug Name	Status
	Renin-Angiotensin System Agonists	RASRx	RASRx1902	Preclinical
	Ang(1-7)/Mas	Tarix	TXA127	Preclinical
	Anti-fibrotic	Akashi	HT100	Discontinued
	Anti-fibrotic	FibroGen	Pamrevlumab	Clinical

Anti-Inflammatory Therapy

	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	Approved

Cardiac Function

	Research	Company	Drug Name	Status
	Cardiac derived stem cells	Capricor	CAP-1002	Clinical
	VIP Agonist	PhaseBio	PB1046 Vasomera	Preclinical
	Membrane sealant	Phrixus	P188	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
	Exon Skipping	BioMarin	Drisapersen Exon 51	Discontinued
	Duplication Exon Skipping	Nationwide Children’s	rAAV.U7 snRNA Exon 2	Preclinical
	Exon Skipping – PMO	Sarepta	Exondys51 Exon 51	Accelerated Approval
	Exon Skipping – PMO	Sarepta	Golodirsen Exon 53	Clinical
	Exon Skipping – PMO	Sarepta	Casimersen Exon 45	Clinical
	Exon Skipping – PMO	Sarepta	Exon 52	Preclinical
	Exon Skipping – PPMO	Sarepta	SRP-5051 Exon 51	Clinical
	Exon Skipping – PPMO	Sarepta	Exon 44	Preclinical
	Exon Skipping – PPMO	Sarepta	Exon 45	Preclinical
	Exon Skipping – PPMO	Sarepta	Exon 50	Preclinical
	Exon Skipping – PPMO	Sarepta	Exon 52	Preclinical
	Exon Skipping – PPMO	Sarepta	Exon 53	Preclinical
	Exon Skipping	Synthena	Tricyclo-LNA	Preclinical
	Stereopure antisense oligonucleotide	Wave	Suvodirsen Exon 51	Clinical
	Stereopure antisense oligonucleotide	Wave	Exon 53	Preclinical
	Exon Skipping	Genethon	rAAV.U7 snRNA Exon 53	Preclinical
	Exon Skipping	NS Pharma	Exon 53	Clinical

Gene Correction

	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

Mitochondrial

	Research	Company	Drug Name	Status
	Coenzyme Q10 analog	Santhera	Idebenone	Clinical
	Mediated gene regulation	Mitobridge	MA-0211	Clinical

Muscle Regeneration

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

Protein Replacement

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

Stop Codon Read-Through

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