Medicine face
Q1 · both planes filled
DMD
Two planes on one gene. Disagreement is the reading, not a hole to fill.A teaching case. Both the canine evidence plane and the human medicine plane hold a row.
Canine plane
Coverage
dark · below bar
lit_any
Lookup · Discovery · Frontier
Lookup abstained (human P/LP signal exists below the 3-star expert-review bar; moderate-confidence anchor (Landrum et al. 2018)). Discovery recovered known. A candidate never fills a drug row. Frontier cause below bar.
Medicine plane
Mechanism
Anchors the extracellular matrix to the cytoskeleton via F- actin.
Full function description, 3 more sentencesFold the description
- Ligand for dystroglycan.
- Component of the dystrophin-associated glycoprotein complex which accumulates at the neuromuscular junction (NMJ) and at a variety of synapses in the peripheral and central nervous systems and has a structural function in stabilizing the sarcolemma.
- Also implicated in signaling events and synaptic transmission.
Source UniProt via Open Targets · PMID 16710609
Formation of the dystrophin-glycoprotein complex (DGC) · Striated Muscle Contraction · Non-integrin membrane-ECM interactions · Reactome
Molecules
Elevidys, generically Delandistrogene Moxeparvovec-Rokl
APPROVALEXOGENOUS GENE · Action on the human target as recorded by the cited medicine-plane source. Not a canine treatment claim and not a disease-direction claim.
Amondys 45, generically Casimersen
APPROVALPOSITIVE MODULATOR · Action on the human target as recorded by the cited medicine-plane source. Not a canine treatment claim and not a disease-direction claim.
Labeled for Duchenne muscular dystrophy.
Drisapersen
PHASE 3POSITIVE MODULATOR · Action on the human target as recorded by the cited medicine-plane source. Not a canine treatment claim and not a disease-direction claim.
Labeled for Duchenne muscular dystrophy.
Eteplirsen
APPROVALPOSITIVE MODULATOR · Action on the human target as recorded by the cited medicine-plane source. Not a canine treatment claim and not a disease-direction claim.
Labeled for Duchenne muscular dystrophy.
More molecule rows are held. This face does not print an approved-drug count.
Canine trials
unqueryable
We hold no canine registry row. The AVMA Veterinary Clinical Trials Registry is the index that would hold one. This station is unqueryable until that ingest exists. That is not evidence that nobody runs trials in dogs.
PK / tox station
stub
This station is not wired. No PK or tox numbers.