Autobahn Therapeutics has successfully completed a pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) regarding the ... Read more
EUnetHTA, Europe’s health technology assessment network, is seeking input from children and adolescents, ages 18 and younger, with early cerebral adrenoleukodystrophy ... Read more
High doses of the B vitamin biotin restored metabolic balance and rescued nerve degradation in a mouse model of adrenoleukodystrophy (ALD), ... Read more
Clinical trials testing Minoryx Therapeutics‘ leriglitazone (MIN-102), an investigational oral therapy for all forms of X-linked adrenoleukodystrophy (ALD) — including childhood ... Read more
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with ... Read more