The pivotal trial for Neurogene's Rett syndrome gene therapy makes use of baseline controls and a rigorous endpoint that ...

More than two-thirds of clinical trials fail to meet original patient enrolment targets, 85% fail to retain enough patients, and the average dropout rate across all trials is 30%. 1 Yet the ...

Monotherapy efficacy and safety profile provided backbone for clinical development strategy in 1L combination with nivolumab plus chemotherapy ...

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Phase 3 study met primary and most key secondary clinical endpointsThis marks the third developmental and epileptic encephalopathies (DEE) population for ...

Targeted therapies and immunotherapy can provide benefits when added to chemotherapy or may eliminate the need for ...

Recently, alternative endpoints, including progression-free survival (PFS), have emerged as popular readouts for many phase 3 clinical trials. PFS indicates the time from diagnosis (or treatment ...

A clinical trial involves the study of the safety, efficacy and/or dosage regimen of a therapeutic intervention (such as a drug) in humans selected according to predetermined criteria of ...