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PTC Therapeutics is seeking approval of PTC-AADC, a gene therapy candidate to treat aromatic l-amino acid decarboxylase (AADC) deficiency, from the European Medicines Agency (EMA), the company announced in a corporate update. Its application is among the highlights from 2019 that the company will present at…
PTC Therapeutics‘ gene therapy candidate PTC-AADC (formerly AGIL-AADC) provided clinically meaningful and sustained improvements in motor, cognitive, and language milestones in children with aromatic l-amino acid decarboxylase (AADC) deficiency up to five years following the one-time treatment, trial analyses show. A single dose of PTC-AADC delivered into…
Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term…
Pakistan has a high rate of inherited metabolic disorders (IMDs) and needs better facilities and resources for improving the screening, diagnosis, and genetic counseling of families across the country, a nationwide screening study says. In two years, 88 cases were confirmed of organic acidurias (OA), aminoacidopathies…
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