| Abstrakt: |
A recent report from the David Geffen School of Medicine at the University of California, Los Angeles (UCLA) discusses the safety and efficacy of gene replacement therapy for X-linked myotubular myopathy, a rare and life-threatening muscle disease. The study involved a multinational, open-label, dose-escalation trial conducted at seven medical centers in Canada, France, Germany, and the USA. The researchers found that most children who received gene replacement therapy experienced improvements in ventilator dependence and motor function, with some achieving ventilator independence and the ability to walk independently. However, there were several deaths reported, and investigations into the risk of hepatobiliary disease and the need for liver function monitoring are ongoing. [Extracted from the article] |
