Already offering the only non-invasive treatment for SMA in liquid form, Roche’s Evrysdi now comes as a tablet.

Dr. Tejal Aslesh Tailor is an accomplished neuroscientist and molecular biologist with years of expertise in preclinical ...

Cell and gene therapy (CGT) represents the pinnacle of biomedical innovation, offering unprecedented potential to treat, ...

Concern about the safety of gene therapies has been thrust into the spotlight again with the news that two patients treated with Novartis' spinal muscular atrophy (SMA) treatment Zolgensma died as ...

Meanwhile, access to Zolgensma in the UK has been suspended in children older than 12 completed months, as there have been a "small number" of cases of liver-related side effects among children ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation ...

Here, the authors have mapped the antibody response of the adeno-associated virus 9 (AAV9) gene therapy vector Zolgensma. AAV9 capsid variants were designed to escape this response while ...

Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

Genetic carrier screening is conducted to determine if an asymptomatic person is a carrier for a potentially inheritable pathogenic gene.