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

Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3).

Orphanet journal of rare diseases - 22 May 2015

Weinreb Neal J, Finegold David N, Feingold Eleanor, Zeng Zhen, Rosenbloom Barry E, Shankar Suma P, Amato Dominick

Abstract excerpt

BACKGROUND: GD1-DS3 is an integrated assessment of type 1 Gaucher disease (GD1) burden based on bone, hematologic and visceral domains. We investigated this disease severity scoring system (DS3) methodology for initial assessment, long-term follow-up and evaluation of treatment responses. METHODS: We enrolled 133 treated adult GD1 patients. Baseline DS3 scores were calculated near the initial treatment date and...

Read the complete abstract on PubMed

Topics

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

Share to Adolescent
Share to Aged

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Alleles

Share to Child, Preschool

Share to Gaucher Disease

Share to Homozygote

Share to Young Adult

Source

Open in PubMed Open full text

Identifiers

PMID: 25994334
DOI: 10.1186/s13023-015-0280-3
PMCID: PMC4471923

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026. Manifest publication-graph-cohort-2026-09-05.1.

Source receipt: pubmed, retrieved 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3).PMID 25994334Read ↗

A validated disease severity scoring system for adults with type 1 Gaucher disease.Semantic proximity Read ↗

Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS).Semantic proximity Read ↗

Baseline characteristics of 32 patients with Gaucher disease who were treated with imiglucerase: South African data from the International Collaborative Gaucher Group (ICGG) Gaucher Registry.Semantic proximity Read ↗

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.Semantic proximity Read ↗

In-Depth Phenotyping for Clinical Stratification of Gaucher DiseaseSemantic proximity Read ↗

Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.Semantic proximity Read ↗

A new severity score index for phenotypic classification and evaluation of responses to treatment in type I Gaucher disease.Semantic proximity Read ↗

Gaucher disease types 1 and 3: Phenotypic characterization of large populations from the ICGG Gaucher Registry.Semantic proximity Read ↗

Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes.Semantic proximity Read ↗

Clinical-genetic characteristics and treatment outcomes of Turkish children with Gaucher disease type 1 and type 3: A sixteen year single-center experience.Semantic proximity Read ↗

The diagnosis and management of Gaucher disease in pediatric patients: Where do we go from here?Semantic proximity Read ↗

3 Gaucher's disease: clinical features and natural historySemantic proximity Read ↗

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