Author Archives: Nisha Cooch, PhD

 While the entire world has been brought to a standstill by the COVID-19 pandemic, many people reacting – rationally and irrationally – to the threat of the virus are healthy, young, and seemingly at low-risk for long-term or dire health consequences. Those with SMA, on the other hand,…

March has kicked off with new SMA publications largely focused on understanding the disease at the cellular and molecular levels. New reviews on progress in SMA treatments have also been released, as has new patient-focused information including new data on carrier frequency. Understanding SMA The clinical spectrum of BICD2 mutations.1…

February wrapped up with a surge of data on SMA, including novel investigations into how combinatorial therapies may provide new advantages to those with the disease. More information on how nusinersen impacts patients has also become available, as have new findings related to newborn screening and the genetics underlying SMA.

Perhaps the biggest SMA news this month has been the success of Roche’s Sunfish trial, but fundamental research, such as those into the mechanisms of the disease and the implications of certain interventions, continue.  Below is a roundup of the latest news and literature on SMA. Understanding SMA Survival…

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.

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…

[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…

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…

Data on SMA continue to roll out. This month has shown a growing focus and understanding on issues related to nusinersen as well as newborn screening for SMA across the globe. In addition, more preclinical and translation results have been published.  Below is the latest in SMA news and…