Spinal Muscular Atrophy: The Functional Improvements That Matter Most to Patients
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YouTubeCheckRare
Spinal Muscular Atrophy: The Functional Improvements That Matter Most to Patients
Alicia Henriquez, MD, Pediatric Neuromuscular Specialist, Seattle Children’s Hospital, offered that seemingly minor improvements in existing SMA motor function scales may actually represent significant improvements in activities that are important to patients and their caregivers. Today’s targeted treatments and gene therapies for SMA are a ...
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