The cost burden of patients with SMA is considerable, and is estimated to be approximately $4 million to $5 million over 10 years in patients with early-onset SMA. This cost is 54.2 times greater than ...

In an exclusive interaction with Professor Philip J Young from the University of Warwick we understood the importance of ...

The effects allowed these minors who could sit but not stand to move like they've never done before, including walking and ...

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...

The U.S. Food and Drug Administration has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of spinal ...

编者按：全世界大约每6000~10000名新生儿中就有1人患有脊髓性肌萎缩症（SMA）。SMA致残、致死率高，一度无药可治。近年随着产业界的不懈努力，多款新药接连问世，患者的生活质量大幅提高。为了实现更高的治疗甚至治愈目标，当下有近百款SMA管线已进入临床研发阶段。作为全球医药创新的赋能者，药明康德一直以来依托一体化、端到端的CRDMO赋能平台，助力全球合作伙伴，推进包括SMA在内的各类疾病的创新 ...

Injections for spinal muscular atrophy (SMA) refer to two treatments that the Food and Drug Administration (FDA) has approved for SMA: nusinersen (Spinraza) and onasemnogene abeparovec-xioi (Zolgensma ...

Spinal muscular atrophy (SMA) is a genetic condition in which the anterior horn motor neurons, as well as other lower motor neurons in the brain stem nuclei, degenerate due to mutations in the ...

Opportunities in the SMA market include leveraging the launch of new myostatin inhibitors, expanding Zolgensma formulations to increase patient eligibility, and capitalizing on cheap nusinersen ...

A preliminary investigation by the Chongqing Municipal Health Commission has found that a doctor at the Chongqing Medical University Children's Hospital injected a child patient with a drug that was ...