Substance / Medication

Migalastat

Overview

Active Ingredient
migalastat
RxNorm CUI
2054252

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Administration details including route, dosing schedules, preparation instructions, and contraindications are pending physician authorship.

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease.
Majid Haya, Verma Neharika, Bhandari Shivani et al. · Expert Opin Pharmacother · 2024
PMID: 38753367Meta-Analysis
Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease.
Lenders Malte, Nowak Albina, Cybulla Markus et al. · Orphanet J Rare Dis · 2025
PMID: 39980015ObservationalFull text (PMC)
Patient reported quality of life and medication adherence in Fabry disease patients treated with migalastat: A prospective, multicenter study.
Müntze Jonas, Lau Kolja, Cybulla Markus et al. · Mol Genet Metab · 2023
PMID: 36709535Observational
Treatment of Fabry Disease management with migalastat-outcome from a prospective 24 months observational multicenter study (FAMOUS).
Lenders Malte, Nordbeck Peter, Kurschat Christine et al. · Eur Heart J Cardiovasc Pharmacother · 2022
PMID: 35512362Observational
Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS).
Lenders Malte, Nordbeck Peter, Kurschat Christine et al. · Clin Pharmacol Ther · 2020
PMID: 32198894Observational
Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data.
Riccio Eleonora, Zanfardino Mario, Ferreri Lucia et al. · Eur J Hum Genet · 2020
PMID: 32647377ObservationalFull text (PMC)
Fabry disease genotype, phenotype, and migalastat amenability: Insights from a national cohort.
Nowak Albina, Huynh-Do Uyen, Krayenbuehl Pierre-Alexandre et al. · J Inherit Metab Dis · 2020
PMID: 31449323Observational
A Kidney Transplant Recipient Treated With Migalastat for Fabry Disease for 33 Months.
Eleftheriadis Theodoros, Divani Maria, Poulianiti Christina et al. · Exp Clin Transplant · 2025
PMID: 40548538Case Report
Challenges in Fabry disease: the combination of two individually amenablevariants may be nonamenable to migalastat.
Fernandes Raquel Menezes, Bento Dina, Marques Nuno et al. · Future Cardiol · 2023
PMID: 36695159Case Report

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Risk profile, common and serious adverse effects, contraindications, and drug interactions are pending physician authorship.

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Migalastat (substance)
SNOMED CT
765340000
UMLS CUI
C2698220
RxNorm CUI
2054252

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.