Neurol Ther April 2023 PMID 36780114

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Claudia A Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S Scalco, Kathryn R Wagner

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Mentions

Peak: 2 in Feb 2023

across 3 sources since Feb 2023

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3,947

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Feb 2023 – Jul 2023

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What people are saying

Discussion examples include mentions of risdiplam's promise as a treatment for SMA patients previously on other therapies, with phrases like 'shows potential' and 'important findings' pointing to their relevance. The tone is analytical but optimistic, focusing on safety, pharmacokinetics, and the impact on patient care, reflecting active engagement and recognition of the study's significance.

AgreementModerate agreement

Most discussions acknowledge the significance of the study results, with some emphasizing the potential of risdiplam for patients with prior treatments.

InterestHigh interest

Posts demonstrate high interest by highlighting new findings and implications for treatment options, indicating active engagement with the research.

EngagementModerate engagement

Comments include references to study methods, safety data, and potential clinical impacts, showing moderate depth of discussion.

ImpactModerate impact

The discussions suggest that the study could influence treatment decisions and future research directions, indicating a moderate to high impact.

Abstract in brief

The JEWELFISH study investigated the safety, tolerability, pharmacokinetics, and pharmacodynamics of risdiplam in 174 pediatric and adult patients with spinal muscular atrophy (SMA) who had previously been treated with other therapies, with results after at least one year of treatment.
Most patients had three copies of the SMN2 gene, and common side effects included respiratory infections and fever, with serious adverse events being relatively rare; the rate of adverse events decreased in the second half of the treatment period.
Risdiplam increased SMN protein levels in blood across all age and weight groups, showing promise as a treatment option even for those with prior therapies, but the study focused mainly on safety and efficacy over a year of treatment.]

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Risdiplam Safety and Efficacy in SMA Patients Previously Treated with Other Therapies

The JEWELFISH study evaluated the safety, tolerability, and pharmacodynamics of risdiplam in 174 pediatric and adult SMA patients previously on other therapies, showing increased SMN protein levels and a generally…

Social RTCL TV · Jul 17, 2023 468 views

Dataemia

@Dataemia · X

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study - https://t.co/oSZKSv2YWy

Feb 22, 2023 · 963 followers View post →

UCL Discovery

@ucl_discovery · X

Feb 22, 2023 · 2,516 followers View post →