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Spinal Muscular Atrophy

Featured Article
Illness costs resulting from spinal muscular atrophy vary widely across disease phenotypes and geographical settings, according to a systematic literature review.
Researchers found that a gene therapy drug for SMA appears efficacious and well tolerated in infants older than 7 months, according to a study published online ahead of print in Pediatric Neurology.

Insights

Shephard Mpofu, MD, senior vice president and chief medical officer, Novartis Gene Therapies, discusses various trials—SPR1NT, LTFU, and RESTORE from Muscular Dystrophy Association Virtual Clinical and Scientific conference—that examined a treatment…
Wildon R Farwell, MD, MPH, global medical head of Neuromuscular Diseases, Biogen, breaks down the treatment challenges and unmet needs in spinal muscular atrophy (SMA), and reviews a recent SMA clinical trial.
Kaliq Chang, MD, discusses common spinal conditions that professionals should be mindful of in older adults and the steps to prevent and treat them.

Interactive Features

According to the National Institutes of Health (NIH), spinal muscular atrophy affects 1 per 8000 to 10,000 people worldwide. Which type of spinal muscular atrophy is the rarest form of the disease?
Which type of spinal muscular atrophy is the most common form of the disease?
Which type of spinal muscular atrophy is characterized by muscle weakness that develops in children aged 6 to 12 months?
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