一个家庭正在筹集数以百万计的资金,为罕见、严重运动障碍的儿童测试基因疗法,等待临床试验。 A family is raising millions to test a gene therapy for children with rare, severe motor disorders, pending clinical trials.

一个家庭正在募集数以百万计的资金来测试一种基因疗法,旨在帮助那些由于罕见遗传条件而患有严重运动障碍的儿童,为那些被困在他们无法移动的身体中的儿童提供可能的救济。 A family is fundraising millions to test a gene therapy aimed at helping children with severe motor impairments caused by rare genetic conditions, offering potential relief for those trapped in bodies they cannot move. 尽管还没有开始临床试验,但这项努力旨在推动为受影响儿童提供试验性治疗办法。 The effort seeks to advance experimental treatment options for affected children, though no clinical trials have yet begun.