DNA replication errors during development are revealed by genomic study.
In May 2019, when the U.S. FDA approved a gene replacement therapy for spinal muscular atrophy (SMA), approval included all children with SMA under age 2. However, the therapy had only been studied in children up to 8 months. A new study looks at the outcomes of a large cohort of patients who have received the therapy since approval.
Diffuse alterations in brain structure and function are seen in youth with epilepsy and symptoms of anxiety or depression.
A prospective, multicenter study following 153 boys younger than 6 years old documents disease progression of Duchenne muscular dystrophy.
A new clinical trial found a cheaper generic drug is effective in protecting the heart in boys with Duchenne muscular dystrophy.
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