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Edition 28 Sep 202618 medical topicsOriginal sources linked
Genetic Diseases
3 articles · Newest source date first
FDA expanded gene-therapy approvalGenetic Diseases ·
CRISPR-based Casgevy expands to children from age two with sickle cell disease or transfusion-dependent β-thalassaemia
The age expansion brings gene-edited autologous stem-cell therapy to much younger children with severe inherited blood disorders.
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Genetic Diseases ·
First gene therapy approved for OTOF-associated inherited hearing loss
Otarmeni uses a dual-AAV system to deliver functional OTOF genetic material directly to inner-ear hair cells.
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Genetic Diseases ·
First approved therapy aimed at neurological manifestations of Hunter syndrome
Avlayah is designed to address a major limitation of conventional enzyme therapy: reaching the central nervous system.
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