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Author

Jian-Jun Gao

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Open access Oct 2026

Rebisufligene etisparvovec: The first AAV9-mediated SGSH gene therapy approved for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA).

Mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome type A) is a rare lysosomal storage disorder caused by biallelic SGSH pathogenic variants that results in heparan sulfate accumulation and progressive neurodegeneration. Previously, treatment was limited to supportive care. In September 2026, the US Food an...

Chen Pang, Yue-Yi Sun, Jian-Jun Gao · 0 citations
Open access Sep 2026

Daraxonrasib: The first-in-class RAS(ON) multi-selective inhibitor approved for treatment of metastatic pancreatic adenocarcinoma.

Advanced pancreatic ductal adenocarcinoma (PDAC) has a poor prognosis, with limited benefits from conventional chemotherapy. Daraxonrasib is an oral, noncovalent RAS(ON) multi-selective inhibitor that forms a ternary complex with cyclophilin A and GTP-bound RAS, blocking downstream signaling across multiple mutant and...

Yue-Yi Sun, Chen Pang, Jian-Jun Gao · 0 citations

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