Evidence map›Paper›PMID 37506209›Full record

ReviewScience advances2023

Mavacamten, a precision medicine for hypertrophic cardiomyopathy: From a motor protein to patients.

Suman Nag, Sampath K Gollapudi, Carlos L Del Rio, James A Spudich, Robert McDowell

Open access · goldAbstract readReview
In one paragraph

Review in Science advances, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 50 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
50citing papers in PubMed, 1 pooled it
15.6field-weighted citation impact, top 1% 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

50 citing papers in PubMed, 1 synthesis or guideline pooled it, 71 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

5 authors at 2 institutions in 2 countries.

Suman Nag *MyoKardia Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA 94005, USA.ORCID 0000-0003-3355-2592
Sampath K Gollapudi *MyoKardia Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA 94005, USA.
Carlos L Del Rio *MyoKardia Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA 94005, USA.ORCID 0000-0002-9761-7888
James A SpudichStanford University School of Medicine, Stanford, CA 94305.ORCID 0000-0002-4324-4254
Robert McDowellMyoKardia Inc., a wholly owned subsidiary of Bristol Myers Squibb, Brisbane, CA 94005, USA.ORCID 0000-0002-4029-5187
Bristol-Myers Squibb (Germany) · DEStanford University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder characterized by left ventricular hypertrophy, hyperdynamic contraction, and impaired relaxation of the heart. These functional derangements arise directly from altered sarcomeric function due to either mutations in genes encoding sarcomere proteins, or other defects such as abnormal energetics. Current treatment options do not directly address this causal biology but focus on surgical and extra-sarcomeric (sarcolemmal) pharmacological symptomatic relief. Mavacamten (formerly known as MYK-461), is a small molecule designed to regulate cardiac function at the sarcomere level by selectively but reversibly inhibiting the enzymatic activity of myosin, the fundamental motor of the sarcomere. This review summarizes the mechanism and translational progress of mavacamten from proteins to patients, describing how the mechanism of action and pharmacological characteristics, involving both systolic and diastolic effects, can directly target pathophysiological derangements within the cardiac sarcomere to improve cardiac structure and function in HCM. Mavacamten was approved by the Food and Drug Administration in April 2022 for the treatment of obstructive HCM and now goes by the commercial name of Camzyos. Full information about the risks, limitations, and side effects can be found at www.accessdata.fda.gov/drugsatfda_docs/label/2022/214998s000lbl.pdf.

Indexed as

Cardiomyopathy, HypertrophicPrecision MedicineBenzylaminesHumansMyosinsUnited StatesUracilBenzylaminesMYK-461MyosinsUracil

Identifiers

PMID37506209
PMCPMC12494088
OpenAlexW4385334933

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.