A Swiss biopharmaceutical company developing treatments for rare and ultra-rare conditions where patients have no approved options.
GAMT deficiency is an ultra-rare inherited metabolic disorder in which the body cannot produce creatine, a molecule essential for energy supply in the brain and muscles. Children born with GAMT deficiency develop intellectual disability, seizures and movement disorders, often within the first years of life.
There is currently no approved drug for this condition. Oral creatine supplementation can restore brain creatine levels and meaningfully improve outcomes when started early. We are working to bring this treatment to patients who need it.
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