Details of Disease

General Information of Disease (ID: DISHFA7H)

• Disease Name: Duchenne dystrophy
• Disease Class: 8C70: Muscular dystrophy
• Disease Hierarchy:
  DIS01GPL: Grass pollen hypersensitivity
  DISHFA7H: Duchenne dystrophy
• ICD Code:
  ICD-11: ICD-11: 8C70
  ICD-10: ICD-10: G71.0

Drug-Interaction Atlas (DIA) of This Disease

This Disease is Treated as An Indication in 6 Approved Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
Casimersen	DMYSNLH	Approved	NA	[1]
Deflazacort	DMV0RNS	Approved	Small molecular drug	[2]
Eteplirsen	DM2QBHP	Approved	Antisense drug	[3]
Golodirsen	DM6NH08	Approved	NA	[4]
Vamorolone	DM047HI	Approved	NA	[5]
Viltolarsen	DMO6YEZ	Approved	NA	[6]

This Disease is Treated as An Indication in 47 Clinical Trial Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
Alisporivir	DM83EBP	Phase 3	Protein/peptide drug	[7]
Angiotensin-(1-7)	DM76GZ0	Phase 3	Small molecular drug	[8]
Ataluren	DM4GXW3	Phase 3	Small molecular drug	[5]
Drisapersen	DMWUN1S	Phase 3	Antisense drug	[9]
Edasalonexent	DMYVBF6	Phase 3	NA	[10]
GSK2402968	DMWL2XD	Phase 3	NA	[11]
HP-802-247	DM6FBGP	Phase 3	NA	[12]
ITF2357	DMFZBNE	Phase 3	Small molecular drug	[5]
Pamrevlumab	DMKTR0A	Phase 3	NA	[5]
RG6206	DMELPGC	Phase 2/3	NA	[5]
Suvodirsen	DMKGQF0	Phase 2/3	Antisense oligonucleotide	[13]
WVE-210201	DMHXJ68	Phase 2/3	NA	[14]
ACE-031	DMZ8PY0	Phase 2	NA	[15]
BMN044	DM3ZFMA	Phase 2	NA	[8]
BMN045	DM2H1SV	Phase 2	NA	[8]
BMN053	DMGOMD1	Phase 2	NA	[8]
CAP-1002	DMJ2Y1C	Phase 2	NA	[16]
CAT 1004	DMQBP7R	Phase 2	NA	[8]
DMD-02	DM4CA2J	Phase 2	NA	[17]
Domagrozumab	DMCT08V	Phase 2	NA	[5]
Gamma-sarcoglycan gene-containing recombinant AAV1 vector based therapy	DMPCSTM	Phase 2	NA	[18]
HT-100	DM2QIN1	Phase 2	Small molecular drug	[17]
MNK-1411	DMICLYJ	Phase 2	NA	[5]
PB1046	DM8MJ3F	Phase 2	Recombinant protein	[19]
PF-06252616	DMQLZOF	Phase 2	Antibody	[8]
PRO-044	DMFYABR	Phase 2	Antisense drug	[20]
SMT-C1100	DMCD6G1	Phase 2	Small molecular drug	[5]
SRP-4053	DMSFI6M	Phase 2	NA	[8]
SRP-5051	DMKRFSA	Phase 2	NA	[21]
SRP-9001	DMR09IH	Phase 2	Gene therapy	[22]
AAV1-FS344	DMAPUZY	Phase 1/2	NA	[5]
BMS-986089	DMO9W4D	Phase 1/2	NA	[8]
EPICATECHIN	DMN0EMP	Phase 1/2	Small molecular drug	[5]
GALGT2 gene therapy	DM1RU0E	Phase 1/2	NA	[5]
PRO-045	DM6GCA1	Phase 1/2	NA	[23]
PRO-053	DMU7M0S	Phase 1/2	NA	[24]
SGT-001	DMYDMVI	Phase 1/2	NA	[5]
Stamulumab	DMBCIXL	Phase 1/2	Antibody	[25]
ARM210	DMQUFNY	Phase 1	NA	[8]
Biostrophin	DMS4P6K	Phase 1	NA	[26]
Carmeseal	DMA05KG	Phase 1	NA	[5]
DT-200	DMYFOJQ	Phase 1	NA	[5]
MA-0211	DMZDMI0	Phase 1	NA	[5]
Micro-dystrophin gene therapy	DMFEUDW	Phase 1	NA	[5]
Muscular dystrophy gene therapy	DM8U4UJ	Phase 1	NA	[27]
MyoDys	DMHO6XP	Phase 1	NA	[28]
PF-06939926	DMUSTOA	Phase 1	NA	[5]

This Disease is Treated as An Indication in 8 Investigative Drug(s)

Drug Name	Drug ID	Highest Status	Drug Type	REF
AMT-080	DMJNNM9	Investigative	NA	[29]
AVI-5038	DMYSBJW	Investigative	NA	[29]
GsMTx4	DMNKYU9	Investigative	NA	[30]
PRO-052	DM1RL26	Investigative	NA	[29]
PRO-055	DM9K17W	Investigative	NA	[29]
PRT-20	DM3HGL9	Investigative	NA	[29]
PRT-300	DM2SJFV	Investigative	NA	[29]
Recombinant human biglycan	DM7WVIC	Investigative	NA	[29]

Molecular Interaction Atlas (MIA) of This Disease

References

• [1] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services. 2021
• [2] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health & Human Services. 2015
• [3] 2016 FDA drug approvals. Nat Rev Drug Discov. 2017 Feb 2;16(2):73-76.
• [4] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services. 2019
• [5] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [6] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services. 2020
• [7] ClinicalTrials.gov (NCT02465203) 3-year Follow-up Study to Assess the Viral Activity in Hepatitis C Patients Who Failed Feeder DEB025/Alisporivir Study.
• [8] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [9] ClinicalTrials.gov (NCT01890798) Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol. U.S. National Institutes of Health.
• [10] ClinicalTrials.gov (NCT03917719) An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD). U.S. National Institutes of Health.
• [11] ClinicalTrials.gov (NCT01480245) Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
• [12] ClinicalTrials.gov (NCT01853384) Safety and Efficacy Trial of HP802-247 in the Treatment of Chronic Venous Leg Ulcers. U.S. National Institutes of Health.
• [13] ClinicalTrials.gov (NCT03907072) Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51). U.S. National Institutes of Health.
• [14] ClinicalTrials.gov (NCT03907072) Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51). U.S. National Institutes of Health.
• [15] ClinicalTrials.gov (NCT01099761) Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
• [16] ClinicalTrials.gov (NCT03406780) A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2). U.S. National Institutes of Health.
• [17] ClinicalTrials.gov (NCT02525302) HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02.
• [18] Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors. Curr Gene Ther. 2012 Jun;12(3):139-51.
• [19] Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
• [20] ClinicalTrials.gov (NCT02329769) Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health.
• [21] ClinicalTrials.gov (NCT04004065) Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM). U.S. National Institutes of Health.
• [22] ClinicalTrials.gov (NCT03769116) A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health.
• [23] ClinicalTrials.gov (NCT01826474) Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy. U.S. National Institutes of Health.
• [24] ClinicalTrials.gov (NCT01957059) A Phase I/II Study of PRO053 in Subjects With Duchenne Muscular Dystrophy (DMD). U.S. National Institutes of Health.
• [25] ClinicalTrials.gov (NCT00104078) Study Evaluating MYO-029 in Adult Muscular Dystrophy. U.S. National Institutes of Health.
• [26] Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800023141)
• [27] Clinical pipeline report, company report or official report of Asklepios BioPharmaceutical.
• [28] Trusted, scientifically sound profiles of drug programs, clinical trials, safety reports, and company deals, written by scientists. Springer. 2015. Adis Insight (drug id 800009280)
• [29] The ChEMBL database in 2017. Nucleic Acids Res. 2017 Jan 4;45(D1):D945-D954.
• [30] URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Ligand id: 4206).