Dosing completed for first three patients in INSPIRE DUCHENNE clinical trial; SGT-003 has been well tolerated in all patients ...

Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company”), a life sciences company developing precision genetic medicines for ...

Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

While investigating a rare developmental disorder, researchers ended up discovering a spectrum of conditions that are all ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with Charcot-Marie-Tooth Disease Type 1 and 2Aarhus, Denmark, 18 November 2024 – NMD ...

Special to Natick Report by Jocelyn Sage Mitchell, Community Outreach Manager at Drink Dion’s Natick and Advisory Board ...

An experimental vaccine showed promise in treating aggressive breast cancer, while a new gene therapy method advances ...