Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD.
Review in American journal of hematology, 2022. 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
0.4field-weighted citation impact, top 44% 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
2 citing papers in PubMed, 3 citations in OpenAlex.
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
12 authors at 6 institutions in 3 countries.
Jonathan D SchwartzRocket Pharmaceuticals, Inc., Cranbury, New Jersey, USA.ORCID 0000-0002-2211-4151
Wilma BarcelliniFoundation IRCCS Ca' Granda Ospedale Maggiore Policlinico Hematology Unit, Pathophysiology of Anemias Unit, Milan, Italy.ORCID 0000-0003-1428-9944
Rachel F GraceDana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0001-7302-0449
Paola BianchiFoundation IRCCS Ca' Granda Ospedale Maggiore Policlinico Hematology Unit, Pathophysiology of Anemias Unit, Milan, Italy.ORCID 0000-0001-5976-5233
Alberto ZanellaFoundation IRCCS Ca' Granda Ospedale Maggiore Policlinico Hematology Unit, Pathophysiology of Anemias Unit, Milan, Italy.
José Luis López LorenzoInstituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain.
Julián SevillaHospital Infantil Universitario Niño Jesús (HIUNJ), Fundación para la Investigación Biomédica HIUNJ, Madrid, Spain.ORCID 0000-0002-6852-1860
Ami J ShahLucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, California, USA.
Bertil GladerLucile Packard Children's Hospital, Stanford University School of Medicine, Stanford, California, USA.
Eileen NicolettiRocket Pharmaceuticals, Inc., Cranbury, New Jersey, USA.
Susana Navarro OrdoñezCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
José Carlos SegoviaCentro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER), Madrid, Spain.
Centre for Biomedical Network Research on Rare Diseases · ESFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico · ITLucile Packard Children's Hospital · USSunesis (United States) · USHarvard University · USHospital Universitario Fundación Jiménez Díaz · ES
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.
Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD. · full record | OpenQuestion