Evidence map›Paper›PMID 42159474›Full record

ArticleAnnals of clinical and translational neurology2026

Evaluation of Digital Technologies for Home-Based Assessment in People With Amyotrophic Lateral Sclerosis.

Arne Mueller, Vanessa Vallejo, Mónica Povedano Panadés, Orla Hardiman, Véronique Danel-Brunaud, Senda Ajroud-Driss, Philippe Couratier, Jeremy Shefner, Anna Gokey, Mike DiCesare and 5 more

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Article in Annals of clinical and translational neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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2 · The registry

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3 · Its place in the literature

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4 · The record

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5 · Who and what money

Authors and funding

15 authors.

Arne MuellerBiomedical Research, Novartis, Basel, Switzerland.ORCID https://orcid.org/0000-0001-6551-2283
Vanessa VallejoBiomedical Research, Novartis, Basel, Switzerland.
Mónica Povedano PanadésBellvitge University Hospital, Barcelona, Spain.
Orla HardimanTrinity College Dublin, Dublin, Ireland.
Véronique Danel-BrunaudHôpital Roger Salengro, Lille, France.
Senda Ajroud-DrissNorthwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
Philippe CouratierCHU de Limoges, Limoges, France.
Jeremy ShefnerBarrow Neurological Institute, Phoenix, Arizona, USA.
Anna GokeyLinus Health, Boston, Massachusetts, USA.
Mike DiCesareZEPHYRx, Troy, New York, USA.
Erin LennoxZEPHYRx, Troy, New York, USA.
Jens PraestgaardBiomedical Research, Novartis, Cambridge, Massachusetts, USA.
Lucie BrujinNovartis Pharmaceuticals UK Ltd, London, UK.
Peggy AllredBiomedical Research, Novartis, Cambridge, Massachusetts, USA.
Ram MillerNovartis Pharmaceuticals Corporation, Cambridge, Massachusetts, USA.

Funding

Novartis Pharma AG
6 · The paper itself

Abstract

objectiveDigital technologies hold promise for transforming healthcare by enhancing personalized treatments and offer valuable opportunities to improve patient care. Here, we evaluated several novel, self-administered, home-based, digital endpoints for their association with corresponding conventional standard clinical measures (primary) in people living with Amyotrophic Lateral Sclerosis (ALS).

methodsThis was a longitudinal study in people with ALS who were followed up to 9 months. A total of 33 participants were enrolled in the study. At each of six visits, participants were evaluated with a battery of conventional standard measurements to determine ALS disease progression and quality of life. Between visits, participants performed weekly home-based self-assessments with digital health technologies (DHT) and self-administered ALSFRS-R. Cross-sectional analysis of DHTs anchored to ALSFRS-R and longitudinal analyses were performed and compared to standard clinical measures.

resultsOf the 33 participants, 20 completed the study, and 13 discontinued before completing the planned 9-month follow-up mainly due to disease progression. The distribution of various digital metrics in home-based assessments corresponded well with the sub-scores of ALSFRS-R in the cross-sectional analyses, with the strongest construct validity for digital speaking rate. In the longitudinal analysis, a weak but significant trend in most metrics was observed, with the strongest trend in the duration of the Timed Up and Go (high variability between participants).

interpretationThe findings from this study provide insights into the potential of digital endpoints to evaluate people living with ALS with the goal of reducing the burden of study participation and improving the efficiency of ALS clinical trials.

Indexed as

ALSFRS‐RALS functional rating scale‐revisedamyotrophic lateral sclerosisdigital endpointshome‐basedself‐administered

Identifiers

PMID42159474
PMCPMC13394927

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