Evidence map›Paper›PMID 38778875›Full record

ReviewTherapeutic advances in rare disease

Understanding patient, caregiver, and healthcare provider perspectives of the management of long-chain fatty acid oxidation disorders.

Eileen Sullivan Baker, Jennifer Botham, Tasia Rechisky, Evelyn Romano, Daniel Garcia, Susan A Berry

Abstract readReview
In one paragraph

Review in Therapeutic advances in rare disease. 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

6 authors.

Eileen Sullivan BakerCaregiver/Advocate, Whitehouse, OH, USA.
Jennifer BothamCaregiver/Advocate, Ottawa, ON, Canada.
Tasia RechiskyPatient/Advocate, Boston, MA, USA.
Evelyn RomanoCaregiver/Advocate, Greenville, SC, USA.
Daniel GarciaMedical Affairs, Ultragenyx Pharmaceutical Inc., 60 Leveroni Ct, Novato, CA 94949, USA.
Susan A BerryUniversity of Minnesota Medical School, Minneapolis, MN, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Long-chain fatty acid oxidation disorders (LC-FAODs) are a group of rare, inherited, metabolic disorders that can lead to a wide range of symptoms that predominantly affect organ systems with high energy needs, such as the heart, liver, skeletal muscle, and nervous system. Clinical management primarily consists of close attention to and monitoring of diet and activity and avoidance of prolonged fasting. In addition, patients and caregivers must be alert for signs of life-threatening metabolic decompensation. As a result, LC-FAODs can have significant and wide-ranging impacts on the lives of patients and their caregivers. This article describes the effects of LC-FAODs at different life stages and in the context of the North American healthcare system from the perspective of a group of patients, caregivers, and healthcare providers (

Indexed as

caregiverlong-chain fatty acid oxidation disordermetabolic conditionpatient

Identifiers

PMID38778875
PMCPMC11110496

What OpenQuestion holds

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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.