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David Cracc @davidcracc ha aggiunto una foto in Health
2025-01-23 07:15:37 · Translate ·

Gene Therapy Market Analysis and Emerging Trends

Introduction Gene therapy has emerged as one of the most transformative innovations in modern medicine, offering potential cures for a range of genetic disorders that were once considered untreatable. By directly targeting the genetic root cause of diseases, gene therapy aims to correct or replace faulty genes, offering patients a chance at long-term recovery and improved quality of...

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David Cracc @davidcracc ha aggiunto una foto in Health
2024-11-28 09:46:27 · Translate ·

PTC Therapeutics' Gene Therapy Receives FDA Nod for AADC Deficiency

PTC Therapeutics has recently received FDA approval for its groundbreaking gene therapy aimed at treating Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency, a rare and debilitating genetic disorder. This approval marks a significant step forward in addressing the unmet medical need for patients with AADC Deficiency, a condition characterized by severe movement and neurological...

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