Spinal Muscular Atrophy

Featured Article
Real-World Trials Show Sustained Durability, Improved Outcomes in Children With SMA
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 for spinal muscular atrophy (SMA), which …
Literature Review Reveals Wide Variability in SMA Costs
Illness costs resulting from spinal muscular atrophy vary widely across disease phenotypes and geographical settings, according to a systematic literature review.
Spinal Muscular Atrophy Drug Appears Effective for Older Babies
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.

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Real-World Trials Show Sustained Durability, Improved Outcomes in Children With SMA
Literature Review Reveals Wide Variability in SMA Costs
Spinal Muscular Atrophy Drug Appears Effective for Older Babies
Study Characterizes Patient, Caregiver Preferences for SMA Treatments
Understanding the Treatment Challenges and Unmet Needs of Spinal Muscular Atrophy
Screening for Spinal Muscular Atrophy Enhances Nusinersen Cost-Effectiveness
Cost of Untreated Spinal Muscular Atrophy is Tremendous, Study Suggests
Identifying a Rare Type of Spinal Muscular Atrophy
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Insights

Real-World Trials Show Sustained Durability, Improved Outcomes in Children With SMA
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…
Understanding the Treatment Challenges and Unmet Needs of Spinal Muscular Atrophy
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.
Caring for the Aging Spine: Pain Management and Preemptive Techniques
Kaliq Chang, MD, discusses common spinal conditions that professionals should be mindful of in older adults and the steps to prevent and treat them.
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Interactive Features

Identifying a Rare Type of Spinal Muscular Atrophy
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?
Common Forms of Spinal Muscular Atrophy
Which type of spinal muscular atrophy is the most common form of the disease?
Link Between Spinal Muscular Atrophy and Muscle Weakness
Which type of spinal muscular atrophy is characterized by muscle weakness that develops in children aged 6 to 12 months?
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