batten-1

Batten-1 is a proprietary pharmaceutical product whose active ingredient is miglustat. It is being developed as an oral therapy for juvenile Batten disease (CLN3 disease), a rare and fatal lysosomal storage disorder affecting the nervous system. Miglustat, the active compound in Batten-1, acts by inhibiting glucosylceramide synthase, thereby blocking the accumulation of glycosphingolipids and reducing neuroinflammation to prevent neuronal death. This mechanism aims to slow or halt progression of neurological symptoms such as motor decline and vision loss. Clinical studies have shown that Batten-1 may stabilize both motor function and visual acuity in patients with CLN3 disease over extended periods of treatment. The drug has demonstrated a favorable safety profile in Phase 1/2 trials, with mostly mild-to-moderate gastrointestinal side effects reported[1][2][3][4][5][6][8].

Brand names

Batten-1Batten1Batten 1

Other names

BBDF 101BBDF101BBDF-101