Researchers Discover Promising New Gene Therapy Approach for Spinal Muscular Atrophy
A new genetic approach to restore the expression of the gene that causes spinal muscular atrophy (SMA) in patients' cells holds promise to effectively and permanently treat the disease, researchers suggest. The study, “Seamless genetic conversion of SMN2 to SMN1 via...
Lake-Breaking SMA Treatment Insights You May Have Missed in February
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...
SMA HCP News December Round-up: Spinal Cord Hypoxia, Scoliosis Surgery & More
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...
Spinal Muscular Atrophy: Late October 2019 Roundup
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...
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