Evidence map›Paper›PMID 37047707›Full record

ArticleInternational journal of molecular sciences2023

High-Dose Ambroxol Therapy in Type 1 Gaucher Disease Focusing on Patients with Poor Response to Enzyme Replacement Therapy or Substrate Reduction Therapy.

Majdolen Istaiti, Dafna Frydman, Tama Dinur, Jeff Szer, Shoshana Revel-Vilk, Ari Zimran

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in International journal of molecular sciences, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
2.6field-weighted citation impact, top 10% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

6 citing papers in PubMed, 15 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 2 countries.

Majdolen IstaitiGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0001-7598-2828
Dafna FrydmanGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Tama DinurGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.
Jeff SzerPeter MacCallum Center, Royal Melbourne Hospital, Department of Medicine, University of Melbourne, Melbourne, VIC 3050, Australia.ORCID 0000-0001-6783-2301
Shoshana Revel-VilkGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0001-9151-0337
Ari ZimranGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0003-0077-8608
Shaare Zedek Medical Center · ILHebrew University of Jerusalem · ILThe Royal Melbourne Hospital · AU

Funding

Takeda (Japan) IISR-2017-104260
6 · The paper itself

Abstract

Ambroxol hydrochloride (ABX), an oral mucolytic drug available over the counter for many years, acts as a pharmacological chaperone for mutant glucocerebrosidase, albeit at higher doses. Proof-of-concept reports have been published over the past decade on all three types of Gaucher disease (GD). Here, we assess the safety and efficacy of 12 months of 600 mg ambroxol per day in three groups of Type 1 GD patients with a suboptimal response to enzyme replacement therapy (ERT) or substrate reduction therapy (SRT), defined as platelet count < 100 × 10

Indexed as

AmbroxolCOVID-19Gaucher DiseaseEnzyme Replacement TherapyHumansLumbar VertebraeAmbroxolambroxolchaperone therapyenzyme replacement therapy (ERT)Gaucher disease (GD)Lyso-Gb1suboptimal responsesubstrate reduction therapy (SRT)

Identifiers

PMID37047707
PMCPMC10095311
OpenAlexW4362634227

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.