The sense of fatigue perceived by patients with spinal muscular atrophy (SMA) does not correlate with their physical tendency to get tired, their motor function, or quality of life, a study reports. According to this…
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Optimal care of children with spinal muscular atrophy (SMA) requires improving competence and an adequate exchange of knowledge among healthcare professionals, as well maintaining a close dialogue with the family, according to a study reporting on the experiences of Swedish and Danish parents. The study, “Bereaved…
Several key factors influence how and why some patients and their families may be hesitant to pursue, or decide to forego, Spinraza (nusinersen) treatment, a study has found. These considerations include side effects, high cost, insurance coverage, and a lack of data on the therapy’s effectiveness. By addressing these…
High-resolution magnetic resonance imaging (MRI) of the spinal cord may be a non-invasive way of monitoring spinal muscular atrophy (SMA) disease progression and responses to treatment, a case report suggests. The technique helped clinicians to diagnose a toddler with SMA type 2 after it revealed signs of ventral nerve atrophy in…
A preliminary Draft Evidence Report from the Institute for Clinical and Economic Review (ICER) says the investigational spinal muscular atrophy (SMA) treatment Zolgensma (onasemnogene abeparvovec-xxxx) could be more cost-effective than Spinraza (nusinersen) with a one-time price of $2 million. However, ICER noted that more must…
SMA News Today brought you daily coverage of important findings, key treatment developments and clinical trials related to spinal muscular atrophy (SMA) throughout 2018. We look forward to reporting more news that is important for the SMA community during 2019. Here are the top 10 most-read SMA articles of 2018, with…
The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of promising therapies by speeding their evaluation so that the therapies, if proven effective, can be available to patients…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
Poland’s Ministry of Health has agreed to pay for Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) regardless of age or disease severity — ending a year-long struggle by the country’s nonprofit Fundacja SMA to gain access to one of the world’s most costly drugs. The surprise announcement…
An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect similar increases in the brain, spinal cord, and peripheral tissues after spinal muscular atrophy (SMA) treatment with the oral therapy risdiplam, researchers say. They believe these findings in animal…
