A recent analysis investigated the state of metabolic disruption experienced by patients with spinal muscular atrophy (SMA), ...
Differences in SMA newborn screening practices in the U.S. could impact referral patterns or the timing of therapeutic ...
Children with spinal muscular atrophy (SMA) experience benefits from receiving a disease-modifying gene therapy, but many ...
Clinical decision-making can be difficult for patients with SMA due to the lack of clinical trials comparing treatment ...
The high cost and limited availability of treatment are still major challenges for patients with a rare genetic disorder ...
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