An evaluation of the combination of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis

Article Clinical Neurology

Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial

Sabrina Paganoni et al.

Summary: Early use of PB/TURSO can prolong tracheostomy/ventilation-free survival in ALS patients and delay the first hospitalization.

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2022)

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Article Clinical Neurology

Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: Evaluating the impact of treatment crossover on outcomes

Sabrina Paganoni et al.

Summary: This study utilized the RPSFTM survival analysis method to analyze data from the CENTAUR trial in ALS, and the results showed a significant survival benefit of PB and TURSO compared to placebo. The findings of this study may contribute to the clinical application of observed survival outcomes in future ALS crossover trials.

MUSCLE & NERVE (2022)

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Review Clinical Neurology

ALSUntangled #64: butyrates

Yuyao Sun et al.

Summary: The article reviews the potential use of butyrates in the treatment of amyotrophic lateral sclerosis (ALS), with a focus on one trial involving sodium phenylbutyrate (NaPB) and Tauroursodeoxycholic acid (TUDCA). The results suggest that this combination can slow ALS progression and prolong survival, but the specific contribution of NaPB is unclear. However, more research is needed to determine the efficacy of butyrates as a treatment for ALS.

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION (2022)

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Article Clinical Neurology

Effects of PB-TURSO on the transcriptional and metabolic landscape of sporadic ALS fibroblasts

Jasmine A. Fels et al.

Summary: This study is the first to explore the molecular effects of AMX0035 in ALS patient-derived cells. It shows that AMX0035 has a greater and distinct impact compared with the individual compounds and provides clues to drug targets and mechanisms of action.

ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY (2022)

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Article Clinical Neurology

Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis

Sabrina Paganoni et al.

Summary: In the CENTAUR trial for ALS, orally administered PB-TURSO significantly slowed functional decline and a long-term survival analysis showed that participants receiving PB-TURSO treatment had a median survival 6.5 months longer than those on placebo. These results suggest that PB-TURSO has both functional and survival benefits in ALS.

MUSCLE & NERVE (2021)

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Article Medicine, General & Internal