News

Nearly half of young people with spinal muscular atrophy (SMA) — and especially those who are underweight — have abnormally high levels of leptin, a metabolic hormone linked to both disease severity and limited mobility, says a new study. The findings show that SMA is associated with abnormal metabolic processes, and researchers suggests…

Cure SMA, an organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), has launched a booklet addressing some of the broader questions about insurance coverage that are common to the SMA community. Decisions can seem overwhelming when faced with a new SMA diagnosis or when caring for…

Children with spinal muscular atrophy (SMA) have poor bone health that leads to common thigh-bone fractures, regardless of the state of their disease, according to a study. Although few of the children could be classified as having osteoporosis, researchers noted that osteoporosis criteria were developed for healthy children. That means the…

Spinal muscular atrophy (SMA) is a step closer to being on a list of diseases that the U.S. government recommends newborns be screened for. Although making the Recommended Uniform Screening Panel list will constitute a powerful statement of support for SMA screening, decisions about what diseases newborns are checked for are…

Fetuses with the most severe form of spinal muscular atrophy (SMA) usually die in the womb, but a newborn with the condition survived 12 days, according to a case study. Infants born with SMA type 0 have mutations of the SMN gene. Since the newborn had fewer copies of the SMN2…

In a recent webinar on Spinraza, the first approved treatment for spinal muscular atrophy (SMA), the patient advocacy group Cure SMA addressed issues such as access and insurance coverage, and answered questions from people affected by the disease. Cure SMA, a foundation that promotes SMA research and provides support for patients and their…

Approximately one in six children and young people with spinal muscular atrophy (SMA) have abnormal sleep, according to a study published in the scientific journal Sleep Medicine. An additional one in six has an abnormal score on at least one sleep factor.

An analysis of U.S. orphan drug designations and approvals suggests that current incentives, detailed in the Orphan Drug Act (ODA) of 1983, have failed to stimulate the development of orphan drugs targeting spinal muscular atrophy (SMA) and other rare diseases. Rather, researchers at the University of Massachusetts-Amherst suggest that the…

Researchers may finally have figured out why a loss in the SMN protein that is crucial for all cells so quickly affects motor neurons, leading infants to develop spinal muscular atrophy (SMA). SMN aids in the transport of certain molecules in all cells, processing RNA, and the mutation-caused drop in its levels within cells…

Access to Spinraza (nusinersen), the first ever approved therapy for the treatment of spinal muscular atrophy (SMA), will be discussed in a webinar tomorrow.