Leriglitazone halts disease progression in adult patients with early cerebral adrenoleukodystrophy (ALD) in compassionate use study published in Brain

In a French early-access program study, 10 of 13 patients were clinically and radiologically stable

Mataró, Barcelona, Spain, 11 June, 2024 - Minoryx Therapeutics (“Minoryx”), a late-stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announce that results from a compassionate use study of leriglitazone for treatment of progressive cerebral adrenoleukodystrophy (cALD) in adult male patients, published1 in the peer-reviewed journal Brain.

The compassionate-use study was led by Fanny Mochel, MD, Ph.D., professor at the Hôspital Universitaire La Pitié-Salpêtrière (Paris, France), as part of an early-access program through the French national drug agency (ANSM) that allows for the use of innovative treatments with favorable safety profiles under a Compassionate Access Authorization protocol (ATU)2. More…