Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease

20 Feb 2025

Abstract excerpt

Abstract Aim The aim of our multicenter study was to investigate the safety and efficacy of enzyme replacement therapy (ERT) and chaperone therapy on the disease progression in female Fabry disease (FD) patients and to compare the individual treatment regimens. Methods Data from 3 consecutive visits of 102 female FD patients from 6 Fabry centers were retrospectively analyzed. According to their FD-specific...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Lysosomal Storage Disorders Research
Share to Whipple's Disease and Interleukins

Source

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Identifiers

PMID: 39980015
DOI: 10.1186/s13023-025-03600-y

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry diseasePMID 39980015Read ↗

Fabry Disease Therapy: State-of-the-Art and Current ChallengesSemantic proximity Read ↗

Fabry disease revisited: Management and treatment recommendations for adult patientsSemantic proximity Read ↗

FABRY DISEASE: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat. What Do We Know Today?Semantic proximity Read ↗

A review and recommendations for oral chaperone therapy in adult patients with Fabry disease.Semantic proximity Read ↗

Treatment of Fabry's Disease With Migalastat: Outcome From a Prospective Observational Multicenter Study (FAMOUS).Semantic proximity Read ↗

Precision medicine in Fabry disease.Semantic proximity Read ↗

A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease.Semantic proximity Read ↗

Developments in the treatment of Fabry disease.Semantic proximity Read ↗

Fabry disease: Mechanism and therapeutics strategiesSemantic proximity Read ↗

The Challenge of Diagnosis and Indication for Treatment in Fabry DiseaseSemantic proximity Read ↗

Long Term Treatment with Enzyme Replacement Therapy in Patients with Fabry DiseaseSemantic proximity Read ↗

Agalsidase-β should be proposed as first line therapy in classic male Fabry patients with undetectable α-galactosidase A activity.Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.