With the onset of the era of technological advancements, it was presumed that numerous challenges, mostly toward the treatment of rare genetic disorders, would be countered. However, the quest for the appropriate treatment continues as one of the rare disorders, Argininosuccinic aciduria (ASA), remains to lack specific treatment or cure. In the past decades, the pharma market has witnessed no new intervention for ASA; thus, the race to bring innovation still prevails.
Argininosuccinic aciduria or argininosuccinate lyase deficiency is one of the inherited disorders belonging to ‘urea cycle disorders,’ which cause ammonia to accumulate in the blood. Six consecutive e...
