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Disease

long QT syndrome 3

Also known as LQT3, SCN5A long QT syndrome, long QT syndrome caused by mutation in SCN5A, long QT syndrome type 3+3 more

LQT3, SCN5A long QT syndrome, long QT syndrome caused by mutation in SCN5A, long QT syndrome type 3, long QT syndrome 2/3, digenic, long QT syndrome 3, acquired, susceptibility to, long QT syndrome 3/6, digenic.

1
Associated genes
1
Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

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Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies
Galsulfaseapproved

Approval — Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confir… (2006)

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2006emaApproval — Galsulfase· Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1). As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. A key issue is to treat young patients aged <5 years suffering from a severe form of the disease, even though patients <5 years were not included in the pivotal phase-3 study. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Associated genes

1 match

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

SCN5A0.84

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Sodium channel protein type 5 subunit alphavia SCN5A0.84

Reference

Authoritative identity, definition & identifiers.

Synonyms

LQT3, SCN5A long QT syndrome, long QT syndrome caused by mutation in SCN5A, long QT syndrome type 3, long QT syndrome 2/3, digenic, long QT syndrome 3, acquired, susceptibility to, long QT syndrome 3/6, digenic

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.