As FDA seeks to rehire some fired employees, Donald Trump threatens to enact tariffs on pharma companies unless they reshore manufacturing; another lawsuit hits the complex GLP-1 compounding space as ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4 ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in all ...

"Jim brings a specialized skillset and innovative technology experience at the start of what will be a year of momentum for ...

Vriddhi, a seven-month-old girl, is battling spinal muscular atrophy type 1, a rare progressive disease that will cause her muscles to weaken over time. The only hope for her survival is a gene ...

New research reveals that voluntary exercise restores STAT3 levels in the amygdala, helping to prevent stress-induced ...

Qure's stock rises on Huntington's therapy progress and strong gene therapy pipeline. Learn about QURE stock's cost-cutting ...

Trial at the Johns Hopkins Kimmel Cancer Center and other centers finds reduced-intensity haploidentical bone marrow ...

Researchers from the NIHR Moorfields Biomedical Research Centre and University College London have found that gene therapy improved visual acuity and preserved retinal structure in young children with ...

Entrada Therapeutics can plough on with the development of its Duchenne muscular dystrophy (DMD) therapy, ENTR-601-44, now ...

When a test determined that Walter had hemophilia, his Omaha owner joined a group seeking to raise $50,000 for a clinical ...

Ten of 11 children born with a rare form of congenital hearing loss experienced improvements after receiving the company’s ...