Evidence map›Paper›PMID 28760888›Full record

ArticleBlood2017

AG-348 enhances pyruvate kinase activity in red blood cells from patients with pyruvate kinase deficiency.

Charles Kung, Jeff Hixon, Penelope A Kosinski, Giovanni Cianchetta, Gavin Histen, Yue Chen, Collin Hill, Stefan Gross, Yaguang Si, Kendall Johnson and 15 more

Open access · bronzeAbstract read
In one paragraph

Article in Blood, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 papers.

0numbers the graph read from it
0cells of the map it votes in
48citing papers in PubMed
3.9field-weighted citation impact, top 6% 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

48 citing papers in PubMed, 125 citations in OpenAlex.

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  6. Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2019
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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

25 authors at 1 institution in 1 country.

Charles KungAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Jeff HixonAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Penelope A KosinskiAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Giovanni CianchettaAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Gavin HistenAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Yue ChenAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Collin HillAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Stefan GrossAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Yaguang SiAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Kendall JohnsonAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Byron DeLaBarreAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Zhiyong LuoShanghai ChemPartner Co., Ltd., Shanghai, China.
Zhiwei GuShanghai ChemPartner Co., Ltd., Shanghai, China.
Gui YaoShanghai ChemPartner Co., Ltd., Shanghai, China.
Huachun TangShanghai ChemPartner Co., Ltd., Shanghai, China.
Cheng FangShanghai ChemPartner Co., Ltd., Shanghai, China.
Yingxia XuShanghai ChemPartner Co., Ltd., Shanghai, China.
Xiaobing LvShanghai ChemPartner Co., Ltd., Shanghai, China.
Scott BillerAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Shin-San Michael SuAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Hua YangAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Janeta Popovici-MullerAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Francesco SalituroAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Lee SilvermanAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Lenny DangAgios Pharmaceuticals, Inc., Cambridge, MA; and.
Agios Pharmaceuticals (United States) · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Pyruvate kinase (PK) deficiency is a rare genetic disease that causes chronic hemolytic anemia. There are currently no targeted therapies for PK deficiency. Here, we describe the identification and characterization of AG-348, an allosteric activator of PK that is currently in clinical trials for the treatment of PK deficiency. We demonstrate that AG-348 can increase the activity of wild-type and mutant PK enzymes in biochemical assays and in patient red blood cells treated ex vivo. These data illustrate the potential for AG-348 to restore the glycolytic pathway activity in patients with PK deficiency and ultimately lead to clinical benefit.

Indexed as

Anemia, Hemolytic, Congenital NonspherocyticAnimalsEnzyme ActivationEnzyme ActivatorsErythrocytesHumansKineticsMicePiperazinesPyruvate KinasePyruvate Metabolism, Inborn ErrorsQuinolinesRecombinant ProteinsSulfonamidesTissue DonorsEnzyme ActivatorsmitapivatPiperazinesPyruvate KinaseQuinolinesRecombinant ProteinsSulfonamides

Identifiers

PMID28760888
PMCPMC5609468
OpenAlexW2739917247

What OpenQuestion holds

Textmetadata
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.