Acetyl-DL-leucine (Tanganil™) in three patients with advanced multiple system atrophy.

ABSTRACT:

Background

Multiple system atrophy (MSA) is a rare, rapidly progressive alpha-synucleinopathy characterized by autonomic dysfunction, parkinsonism and marked cerebellar ataxia, with very limited treatment options. N-acetyl-DL-leucine (ADLL) (Tanganil™), a racemic derivative of leucine is usually well tolerated and has shown promise in improving cerebellar symptoms and reducing REM sleep behavior disorder (RBD), a common non-motor dream-sleep disorder feature of alpha-synucleinopathy. Given this dual potential, we treated three patients with advanced-stage MSA under compassionate use rules.

Case presentations

Three patients with advanced-stage MSA and severe cerebellar symptoms-two of whom also had moderate RBD-were treated with ADLL (Tanganil^TM), titrated to 5g/da

SUBMITTER: Oertel WH 

PROVIDER: S-EPMC12490026 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature