A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

NVS' phase III study on experimental gene therapy candidate Intrathecal onasemnogene abeparvovec (OAV101IT) meets its primary ...

Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle ...

On 30 December 2024, Novartis reported that its intrathecal (IT) formulation of onasemnogene abeparvovec (OAV101 IT) reached ...

I recently watched an episode of one of my favourite TV shows, the Good Doctor. The episode revolved around how a man named Wyatt was rushed into the ER as he had a perforated valve and needed ...

Among the FDA’s pending decisions for this quarter are Vertex’s non-opioid pain drug and Sanofi’s RNA interference therapy ...

Bryan Johnson, who’s spending hundreds of millions of dollars on treatments designed to extend his life, is many things to ...

For instance, the patients receiving them have to fully understand the procedure, its risks, its advantages and the ...

This interview comes at the team celebrates its 100 th retinal gene therapy procedure. 1 Since 2018, Nagiel and his team at the Vision Center of CHLA have been treating these patients with the gene ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

The Congressional Budget Office said policies to increase treatment uptake could affect Medicaid, Social Security, and other government programs.

The phase 3 STEER study enrolled 127 treatment-naïve patients aged 2 to less than 18 years with SMA type 2 who were able to sit but had never walked independently.