News

Research on SMA continues to forge ahead in 2020, with new results in from Roche’s latest investigation into its oral SMA drug, risdiplam. New tools for screening are being revealed as legislative changes are pushing more and more toward requiring infants born in the U.S. to be screened for SMA.

Patients with SMA often spend the majority of their time either in their wheelchair or in their bed. Since they can be fairly limited to these two places, it is important to increase their independence within those areas. Although my patient with SMA, Michael, will most likely not be able…

New SMA research and news related to newborn screening and the upcoming FDA evaluation of a new drug have kicked off 2020. Below is a roundup of the latest on SMA. Understanding SMA Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy.1  This paper describes a study…

Understanding SMA Stasimon contributes to the loss of sensory synapses and motor neuron death in a mouse model of spinal muscular atrophy.1 In this paper, the authors elucidate a mechanism of the motor neuron degeneration that occurs in SMA. Specifically, the authors found that dysfunction of Stasimon, a protein of…

2019 was a year of significant progress in our understanding of SMA and how to best address it – clinically and otherwise. Below is a roundup of some of the final SMA data shared in late 2019. Understanding SMA The classical complement pathway mediates microglia-dependent remodeling of spinal motor circuits…

With patients that are diagnosed with SMA, they can have a hard time controlling their neck and trunk, which makes it more difficult to be independent with chair mobility, such as ascending and descending ramps, as well as assisting with transfers. When first starting these exercises, it is…

[et_pb_section admin_label=”section”] [et_pb_row admin_label=”row”] [et_pb_column type=”4_4″][et_pb_text admin_label=”Text”]Several new papers on the mechanisms underlying SMA have been published in recent weeks, and data on pertinent SMA treatments continue to be collected and shared. Importantly, researchers also continue to consider how the science and medicine around SMA can best be…

Regulators continue to review SMA drugs and consider policies related to newborn screening while new data emerge related to the mechanisms underlying the disease, as well as those concerning management and treatment.  Here is a roundup of some of the latest news and research related to SMA. Understanding SMA…

Working with a patient with SMA is not all that different from working with any other patient. Your plan of care advances and changes while working with the patient and making observations as you see fit to the patient’s overall wellbeing. To get Michael positioned for the assisted…

While the biggest news this week may be the success of the drug risdiplam in improving motor function in a late stage clinical trial on patients with SMA types 2 and 3,1 other important data on SMA have also been published in recent weeks. Below is a roundup of new…