Subsequent studies by other investigators confirmed those early results, ultimately leading to the FDA approval of ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

SpliceBio has closed a Series B financing round, raising $135m for the clinical development of SB-007 for Stargardt disease.

Panelists discuss how emerging intrathecal gene therapy for older patients with spinal muscular atrophy (SMA) shows modest ...

Spacecraft unveils 1st-ever images of sun's south pole: 'Beginning of a new era' ...

Health secretary Robert F. Kennedy Jr. yesterday appointed a new, smaller vaccine advisory committee at the CDC — just two ...

"FDA approves Capsida’s CAP-003 gene therapy for Parkinson’s" was originally created and published by Pharmaceutical ...

The FDA’s accelerated approval pathway plays a vital role, and scrapping it or reversing course on already-approved therapies ...

The benefits of drug warranties extend beyond risk management. They can also serve as market differentiators in crowded ...

Delcath Systems ( NASDAQ: DCTH) is a company focused on HEPZATO, a treatment for certain forms of metastatic uveal melanoma, ...