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Advancing Potency Assay Development for Advanced Therapy Medicinal Products: A Comprehensive Approach and Regulatory Insights Alaa Abdellatif, Melissa Bou Jaoudeh, Alex Zwiers, and Gabrièle Breda https://www.liebertpub.com/doi/10.1089/hum.2024.249?utm_campaign=MAL_HUM&utm_medium=email&_hsenc=p2ANqtz-9gXPsv4rGkc2mOU4et9-o_m5kwwvRxiSEt2t1k1zsoiJ3s4VbEWkEBuO4pkq75ctR6G9ts1AxzKEyiMtcn8hAYbMmACA&_hsmi=390845464&utm_content=390845464&utm_source=hs_email The Lived Experience of Pediatric Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: Exploring Perceptions of Parents and Professionals Using Social Representation Method Shotaro Tachibana, Dominique Vincent-Genod, Pascal Rippert, Carole Vuillerot, and Silvana De Lucia https://www.liebertpub.com/doi/10.1177/10430342251359998?utm_campaign=MAL_HUM&utm_medium=email&_hsenc=p2ANqtz--niSjfFQjaMTocSBMIN3NawJANsMv9YqGzxdiSWSJDAZ59M_YzTzZhUez3TjzxzTZhRuXroVEcwB3A1WTVswVNDrRJgA&_hsmi=390845464&utm_content=390845464&utm_source=hs_email Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis-Regulatory Elements Quang Hong Pham, Venkata Anudeep Bheemsetty, Phuong Anh Nguyen, Ermira Samara-Kuko, Fangye Gao, Marinee K. Chuah, and Thierry VandenDriessche https://www.liebertpub.com/doi/10.1177/10430342251359989?utm_campaign=MAL_HUM&utm_medium=email&_hsenc=p2ANqtz--XFOpn1pT0Anb6oJ5fYZXmgL_x9FGavN2ioG0JNMtwKbhXdjHwy-OkxEooJNDfxWEEAwHPUEpNQl9zbjyRUiFJecFhig&_hsmi=390845464&utm_content=390845464&utm_source=hs_email