Evidence map›Paper›PMID 41710743›Full record

ReviewACS pharmacology & translational science2026

Advances in Pharmacological Treatments for Cystinosis: Cysteamine and Its Alternatives.

Aitor Carneiro, D Heulyn Jones

Abstract readReview
In one paragraph

Review in ACS pharmacology & translational science, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
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

2 citing papers in PubMed.

  1. Article
  2. Article
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

2 authors.

Aitor CarneiroMedicines Discovery Institute, School of Biosciences, Cardiff University, Main Building, Park Place, Cardiff CF10 3AT, U.K.ORCID https://orcid.org/0000-0002-7415-5588
D Heulyn JonesMedicines Discovery Institute, School of Biosciences, Cardiff University, Main Building, Park Place, Cardiff CF10 3AT, U.K.ORCID https://orcid.org/0000-0002-4290-2999

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Cystinosis is an inherited lysosomal storage disorder characterized by the intralysosomal accumulation of crystals of cystine. This alteration is caused by the absence of the lysosomal membrane transporter cystinosin, which leads to clinical manifestations of the disease. Oral administration of aminothiol cysteamine, while not a curative therapy, has proven to be effective in controlling the progress of the disease and reducing its complications. However, the numerous side effects inherent to the treatment are responsible for low patient compliance, severely impacting therapy success. Several studies have been performed in the past few years with the aim of optimizing cysteamine therapy to avoid its main drawbacks. This review focuses on the potential and feasibility of these novel strategies. As well, it introduces novel recent approaches studied as an alternative or complement to cysteamine treatment.

Indexed as

cysteaminecystinecystinosincystinosislysosomesrare diseases

Identifiers

PMID41710743
PMCPMC12910489

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.