Glycogen storage disease due to acid maltase deficiency
Also known as Alpha-1,4-glucosidase acid deficiency, GSD due to acid maltase deficiency, GSD type 2, Glycogen storage disease type 2+3 more
Alpha-1,4-glucosidase acid deficiency, GSD due to acid maltase deficiency, GSD type 2, Glycogen storage disease type 2, Glycogenosis due to acid maltase deficiency, Glycogenosis type 2, Pompe disease.
Recent clinical, regulatory, research and industry developments relating to this disease.
Marketing authorisation: Nexviadyme (EMA)
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Original application approval — Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combin… (2023)
Original application approval — Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme repl… (2023)
Original application approval — Nexviadyme (avalglucosidase alfa) is indicated for long-term enzyme replacement therapy f… (2022)
Original application approval — Myozyme is indicated for long-term enzyme-replacement therapy (ERT) in patients with a co… (2006)
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
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
Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.
Reference
Authoritative identity, definition & identifiers.
Alpha-1,4-glucosidase acid deficiency, GSD due to acid maltase deficiency, GSD type 2, Glycogen storage disease type 2, Glycogenosis due to acid maltase deficiency, Glycogenosis type 2, Pompe disease
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Related entities are derived from literature co-mention (studied together) — associative, not causal.