Evidence map›Paper›PMID 40965373›Full record

ArticleEuropean journal of neurology2025

Real-World Clinical Experience With Sodium Phenylbutyrate and Taurursodiol at a Single Amyotrophic Lateral Sclerosis Center in the United States.

Narghes Calcagno, Erica Scirocco, Erin Clampffer, Grace Addy, Sean Morgan, Neel Parikh, Dylan V Neel, Jennifer Scalia, Rebekah Young, Matteo Locatelli and 3 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in European journal of neurology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
–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

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Review
  3. Article
  4. Article
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

13 authors.

Narghes CalcagnoSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0003-0615-891X
Erica SciroccoSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0009-0002-9427-1452
Erin ClampfferSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Grace AddySean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Sean MorganSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Neel ParikhSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Dylan V NeelSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0001-6964-9842
Jennifer ScaliaSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Rebekah YoungAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.
Matteo LocatelliSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
deMauri S MackieAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, USA.ORCID 0000-0002-4941-1682
James D BerrySean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0003-0898-6081
Sabrina PaganoniSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID 0000-0003-0505-1168

Funding

Amylyx Pharmaceuticals Inc
6 · The paper itself

Abstract

INTRODUCTION/

aimsSodium phenylbutyrate (PB) and taurursodiol (TURSO)-PB and TURSO-was approved as a treatment for amyotrophic lateral sclerosis (ALS) in the United States in 2022 based on the results of a phase 2 trial, but was voluntarily withdrawn from the market in 2024 after negative results from its phase 3 trial. The objective of our study was to describe the real-world clinical experience with PB and TURSO at one ALS clinic.

methodsThis was a retrospective chart review of all ALS patients receiving a PB and TURSO prescription at the Massachusetts General Hospital ALS Clinic between October 1, 2022, and September 30, 2023. Data were extracted from patients' electronical medical records up to December 31, 2023.

resultsA total of 441 ALS patients received a PB and TURSO prescription, with 329 (75%) initiating the medication. The average length of treatment was 285 days, and the rate of drug discontinuation during the study period was 41% (N=135). The most common reason for drug discontinuation was side effects (N=93, 69%), with the most common being gastrointestinal issues (N=69). No hospitalizations, deaths, or serious adverse events were considered related to treatment with PB and TURSO. DISCUSSION: Experience in real-world clinical settings can help supplement trial data with information on the drug performance at various stages of disease progression. Adverse events impacted treatment persistence in routine clinical practice, underscoring the need for vigilant monitoring and tailored supportive interventions to optimize treatment adherence.

Indexed as

Amyotrophic Lateral SclerosisPhenylbutyratesAdultAgedFemaleHumansMaleMiddle AgedRetrospective StudiesUnited States4-phenylbutyric acidPhenylbutyratessodium phenylbutyrate and taurursodiolamyotrophic lateral sclerosisdrug safety profileinsurance constraintsPB TURSOreal‐word experience

Identifiers

PMID40965373
PMCPMC12445112

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Registered trials

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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.