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News about "Rare disease treatment"
Genethon and Ampersand Biomedicines Partner to Develop Precision Gene Therapy Vectors
Genethon and Ampersand Biomedicines join forces to engineer advanced AAV gene therapy vectors designed for greater tissue specificity, potentially enabling safer and more effective treatments for patients with rare genetic disorders.
Rare Disease Treatment | 25/05/2026 | By News Bureau
Regenxbio Reports Positive Pivotal Trial Results for Duchenne Gene Therapy RGX-202
Regenxbio’s investigational gene therapy RGX-202 demonstrated strong microdystrophin expression, functional gains and a favourable safety profile in Duchenne muscular dystrophy patients, supporting plans for accelerated approval and a potential commercial launch in 2027.
Rare Disease Treatment | 15/05/2026 | By News Bureau
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