Clinical Studies and Trials

NEAT

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This trial will evaluate the effects of EryDex in patients with A-T.

Global A-T Family Data Platform

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The Platform is a patient-driven effort through which data about people with A-T are shared with researchers.

Pulmonary Function Study

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This study seeks to establish the natural history of pulmonary function decline in the A-T population.

Nicotinamide Riboside (Niagen) Proof-of-concept Study

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This trial in the Netherlands looked at the effects of nicotinamide ribose as a potential treatment for people with A-T.

PET Scan/FMRI Study

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Participants with A-T demonstrated lower brain activity or metabolism in the cerebellum as compared to control participants.

Baclofen Study

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At the dose tested, Baclofen did not have a significant effect on tremor, eye movement or balance deficits.

Dexamethasone Trial

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This open label study was performed to assess the safety and tolerability of the EryDex System in people with A-T.

Betamethasone Trials

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These trials studied whether a low dose betamethasone would reduce neurological symptoms associated with A-T.

Effect of Amantadine on Movement Disorder in A-T

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This trial was performed to see if amantadine sulphate improves ataxia or other movement disorders associated with A-T.

Cerebrospinal Fluid Biomarker Study

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Among the 204 identified CSF proteins, 13 showed significant changes in A-T patients versus control samples.