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Strengthening health systems for access to gene therapy in rare genetic disorders

13 Mar 2024

Abstract excerpt

After decades of innovation, the promise of gene therapy is becoming a reality for patients with certain rare genetic disorders. Several cell and gene therapies have already been approved by regulators worldwide,1Landfeldt E. Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives.J. Neuromuscul. Dis. 2022; 9: 675-688https://doi.org/10.3233/JND-221540Crossref PubMed Scopus (2)...

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PMID: 38516694
DOI: 10.1016/j.omtm.2024.101220

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