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Muscle Hypotonia clinical trials

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NCT ID: NCT05911399 Available - Clinical trials for Allan-Herndon-Dudley Syndrome

Expanded Access Program for Tiratricol in Patients With Monocarboxylate Transporter 8 Deficiency

Start date: n/a
Phase:
Study type: Expanded Access

The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS]), who in their Treating Physician's opinion, could benefit from tiratricol and meet the eligibility criteria.