The Amsterdam-based biotech firm said that its melt curve analysis-based test is used as a first-tier genetic screening tool to aid the diagnosis of SMA.

Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...

Scholar Rock submitted its BLA of apitegromab for the treatment of patients with Spinal Muscular Atrophy with a request for ...

A man with a muscle-wasting disease is pleased Manitoba is asking a federal agency to reconsider its opposition to a ...

In a good-news-bad-news week for Biogen, the company will cut an undisclosed number of employees, just as a higher dose of ...

Scholar Rock , a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein ...

Analytics company GlobalData has shared its optimism regarding the future of Novartis’ spinal muscular atrophy (SMA ... met its primary endpoint when testing the intrathecal formulation of ...

MNDs are neurological disorders that destroy motor neurons, the cells that control skeletal muscle activity such as walking, breathing, speaking, and swallowing.

Laboratory tests can monitor for renal toxicity and coagulation abnormalities, including acute severe low platelet counts, which have been observed after administration of some ASOs. Biogen licensed ...

Spinal muscular atrophy (SMA) is a rare hereditary neuromuscular ... in 2022', when a Covid-19 outbreak in Shanghai led to mass testing and strict lockdown conditions. Even more worryingly ...

According to the scientists of the National Research University "BelSU", the obtained results allow us to conclude that the investigated drug provides a pronounced expression of the dysferlin gene and ...

Princeton Biopartners, a best-in-class provider of integrated consulting solutions to the life sciences sector, proudly announces the appointment of Synøve Kamøy, M.D., as Engagement Manager. This ...