Evidence map›Paper›PMID 40946882›Full record

ReviewAmerican heart journal2026

Developing therapeutics for rare cardiovascular diseases.

Joseph B Lerman, Dwight D Koeberl, Shilpi Epstein, Lothar Roessig, Rodica Stan, Meghan Halley, Anjali T Owens, Barry Greenberg, Kevin M Alexander, Sharlene M Day and 5 more

Abstract readReview
In one paragraph

Review in American heart journal, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

15 authors.

Joseph B LermanDuke Clinical Research Institute, Durham, NC. Electronic address: Joseph.Lerman@Duke.edu.
Dwight D KoeberlDivision of Medical Genetics, Department of Pediatrics, Duke University, Durham, NC.
Shilpi EpsteinBayer AG, Wuppertal, Germany.
Lothar RoessigBayer AG, Wuppertal, Germany.
Rodica StanNational Institutes of Health, National Center for Advancing Translational Sciences, Bethesda, MD.
Meghan HalleyCenter for Biomedical Ethics, Stanford University School of Medicine, Stanford, CA.
Anjali T OwensDivision of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
Barry GreenbergDepartment of Medicine/Cardiology, University of California, San Diego, CA.
Kevin M AlexanderDivision of Cardiology, Department of Medicine, Stanford University, Stanford, CA.
Sharlene M DayDivision of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
Mathew S MaurerDivision of Cardiology, Department of Medicine, Columbia University Irving Medical Center, New York, NY.
Eric D AdlerDepartment of Medicine/Cardiology, University of California, San Diego, CA.
Adrian F HernandezDuke Clinical Research Institute, Durham, NC.
Euan A AshleyDivision of Cardiology, Department of Medicine, Stanford University, Stanford, CA.
G Michael FelkerDuke Clinical Research Institute, Durham, NC.

Funding

Genomic Analysis of Network Perturbations in Human DiseaseP50HG004233 · NHGRI · DANA-FARBER CANCER INST · PI VIDAL, MARC · 2007 to 2017
$31.6M
Institutional Career Development Core (KL2)KL2TR003143 · NCATS · STANFORD UNIVERSITY · PI ASCH, STEVEN M. · 2019 to 2023
$8.7M
Postdoctoral Training in Cardiovascular Clinical ResearchT32HL069749 · NHLBI · DUKE UNIVERSITY · PI MARK, DANIEL B · 2003 to 2023
$6.7M
NCATS NIH HHS KL2 TR003143NHGRI NIH HHS P50 HG004233NHLBI NIH HHS T32 HL069749
6 · The paper itself

Abstract

Rare cardiovascular diseases, while individually uncommon, collectively affect millions of people worldwide and are associated with significant morbidity, mortality, and economic burden. Despite this considerable impact, most rare cardiovascular diseases lack approved treatments. Developing therapies for rare cardiovascular diseases requires overcoming a unique set of challenges. This includes barriers to accurate patient diagnosis (and therefore to trial cohort generation), small cohort sizes, the choice of effective clinical trial endpoints, unique ethical and regulatory concerns, and the often-substantial costs of such therapies (which may limit public access to treatment). Despite such challenges, the past decade has witnessed a significant increase in the successful development of rare cardiovascular disease therapies. This review provides an overview of the challenges, while also highlighting potential strategies to advance the field.

Indexed as

Cardiovascular DiseasesRare DiseasesClinical Trials as TopicHumans

Identifiers

PMID40946882
PMCPMC12498264

What Socratic 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 Socratic graph.