nexviazyme
(avalglucosidase alfa)Genzyme Corporation
Usage: NEXVIAZYME is indicated for treating patients aged 1 year and older with late-onset Pompe disease, a condition caused by lysosomal acid alpha-glucosidase (GAA) deficiency.
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pombiliti
(cipaglucosidase alfa-atga)AMICUS THERAPEUTICS US, LLCUsage: POMBILITI, in combination with Opfolda, is indicated for treating adult patients with late-onset Pompe disease (GAA deficiency) weighing ≥40 kg, who are not experiencing improvement on their current enzyme replacement therapy (ERT).sodium phenylbutyrate - sodium phenylbutyrate powder
(Sodium Phenylbutyrate)Endo Usa, Inc.Usage: Sodium phenylbutyrate powder is indicated as adjunctive therapy for chronic management of urea cycle disorders, including neonatal-onset and late-onset deficiencies. It is used to lower ammonia levels in patients, especially those with a history of hyperammonemic encephalopathy, in conjunction with dietary protein restriction.