Disease Information

General Information of the Disease (ID: DIS00276)

• Name: Duchenne muscular dystrophy
• ICD: ICD-11: 8C70

Type(s) of Resistant Mechanism of This Disease

  EADR: Epigenetic Alteration of DNA, RNA or Protein

  IDUE: Irregularity in Drug Uptake and Drug Efflux

Drug Resistance Data Categorized by Drug

Approved Drug(s) 1 drug(s) in total

Deflazacort

Drug Resistance Data Categorized by Their Corresponding Mechanisms

Irregularity in Drug Uptake and Drug Efflux (IDUE)

Key Molecule: ADP/ATP translocase 2 (ADT2) [1]

• Resistant Disease: Duchenne muscular dystrophy [ICD-11: 8C70.1]
• Molecule Alteration: Expression; Up-regulation
• Resistant Drug: Deflazacort
• Experimental Note: Discovered Using In-vivo Testing Model
• In Vivo Model: C57BL10 mouse model; Mus musculus
• Mechanism Description: Deflazacort was found to reduce the resistance of skeletal mitochondria to MPT pore opening, which may be associated with a change in the level of ANT2 and CypD.

Investigative Drug(s) 1 drug(s) in total

Preimplantation factor

Drug Resistance Data Categorized by Their Corresponding Mechanisms

Epigenetic Alteration of DNA, RNA or Protein (EADR)

Key Molecule: H19, imprinted maternally expressed transcript (H19) [2]

• Resistant Disease: Duchenne muscular dystrophy [ICD-11: 8C70.1]
• Molecule Alteration: Up-regulation; Interaction
• Resistant Drug: Preimplantation factor
• Experimental Note: Revealed Based on the Cell Line Data
• In Vitro Model: C2C12 cells; Skeletal muscle; Homo sapiens (Human); CVCL_0188
• In Vivo Model: Mdx mice model; Mus musculus
• Mechanism Description: SPIF promotes myoblast differentiation and utrophin expression while inhibiting fibrosis in Duchenne muscular dystrophy via the H19/miR-675/let-7 and miR-21 pathways.

References

• Ref 1: The Effect of Deflazacort Treatment on the Functioning of Skeletal Muscle Mitochondria in Duchenne Muscular Dystrophy .Int J Mol Sci. 2020 Nov 19;21(22):8763. doi: 10.3390/ijms21228763. 10.3390/ijms21228763
• Ref 2: Downregulation of lncRNA H19 sensitizes melanoma cells to cisplatin by regulating the miR-18b/IGF1 axisAnticancer Drugs. 2020 Jun;31(5):473-482. doi: 10.1097/CAD.0000000000000888.