Genetic renaissance: a cross-regional analysis of the approval landscape of authorised gene therapeutics in paediatrics, challenges and future prospects.

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Title: Genetic renaissance: a cross-regional analysis of the approval landscape of authorised gene therapeutics in paediatrics, challenges and future prospects.
Authors: Ahmad A; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Simran S; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Kalokhe VM; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Musthafa F; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Gangawane VS; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Choudhary KK; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India., Raghuvanshi RS; Central Drugs Standard Control Organization (CDSCO), Ministry of Health & Family Welfare, Government of India, Directorate General of Health Services, New Delhi, India., Srivastava S; Department of Regulatory Affairs, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India. saurabh@niperhyd.ac.in.; Department of Pharmaceutics, National Institute of Pharmaceutical Education and Research (NIPER), Balanagar, Hyderabad, 500037, Telangana, India. saurabh@niperhyd.ac.in.
Source: Journal of community genetics [J Community Genet] 2025 Oct; Vol. 16 (5), pp. 513-528. Date of Electronic Publication: 2025 May 21.
Publication Type: Journal Article
Journal Info: Publisher: Springer Country of Publication: Germany NLM ID: 101551501 Publication Model: Print-Electronic Cited Medium: Print ISSN: 1868-310X (Print) Linking ISSN: 1868310X NLM ISO Abbreviation: J Community Genet Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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ISSN:1868-310X
DOI:10.1007/s12687-025-00799-y