Developed at the Children's Hospital of Philadelphia (CHOP) and called Zynteglo, the medicine uses the patient's own stem ...

Benefits of the gene therapy persisted over several years of follow-up. Presymptomatic children with MLD appeared to have the best outcomes. Severe motor impairment or death was significantly less ...

Department of Polymeric Materials, School of Materials Science and Engineering, Tongji University, 4800 Caoan Road, Shanghai 201804, China Department of Gynaecology and Obstetrics, Shanghai Key ...

Drinking shots gets a whole new meaning for those who order the Bone Marrow and wipe it clean as the marrow turns into a vessel for the shot to be poured into your mouth. Sid Mewara, one half of ...

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne muscular dystrophy, focusing on institutional readiness and patient-centered care. Guidelines ...

Stocktwits - Shares of gene therapy developer uniQure N.V. (QURE) shot up 41% on Thursday afternoon after it announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy ...

Advani (Padma Shri, Padma Bhushan), announced the opening of a Centre of Excellence in CAR T-Cell Therapy and Bone Marrow Transplantation at Smita ... globally benchmarked cell and gene therapies to ...

The Golden Bachelor' Gerry Turner is keeping fans updated on his rare bone marrow cancer. Here's what he said about his life expectancy. Sadly, Turner was diagnosed with Waldenström’s ...

Marks fourth FDA designation for ATSN-201, which has also received Fast Track, Rare Pediatric Disease and Orphan Drug Designations DURHAM, N.C., April 15, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, ...

This study will evaluate SRP-9005, an investigational gene therapy. Additionally, the company has completed enrollment and dosing for Study SRP-9004-102 (DISCOVERY), a phase 1 proof-of-concept study ...