The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.
In a Phase I/II trial, patients with X-linked chronic granulomatous disease will receive a treatment created using a new ...
The firm said it has received positive feedback from the FDA on the design of a Phase III trial of ELI-002 in KRAS-mutant pancreatic cancer.
NEW YORK – Scientists are working to better understand the role of germline pathogenic variants in the development of ductal carcinoma in situ (DCIS) and whether germline variants influence which ...
BH-30643 is an OMNI-EGFR inhibitor that targets classical, atypical, and compound EGFR mutations and acquired resistance mutations in HER2.
HHS is asking the court to affirm its Office of Inspector General's finding that paying for fertility services would run ...
Doctors and patient advocates supporting pretreatment testing at times challenged FDA and other experts who aren't swayed by available evidence.
The company anticipates launching three CRISPR products by 2030, including therapies for hereditary angioedema and ...
The companies will focus on advancing AXV-101, with which they hope to treat retinal dystrophy by targeting mutations in the BBS1 gene.
Despite Leqembi's initial slow sales, the firm's CEO cited areas for the drug's growth and ambitions for an antisense ...
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