Gene Therapy Now Leads Spinal Muscular Atrophy Treatment as Real-World Sequencing Patterns Emerge
A large US claims analysis shows that after widespread newborn screening, gene replacement therapy has become the dominant first treatment ...
A large US claims analysis shows that after widespread newborn screening, gene replacement therapy has become the dominant first treatment ...
A large multicenter study finds that plasma p-tau181, an approved Alzheimer's blood biomarker, is elevated in ALS and rises over ...
A Spanish cohort study finds that low appendicular skeletal muscle mass and sarcopenia independently predict mortality in ALS, with sarcopenia ...
Kyoto University researchers used adenine base editing to correct the TFG mutation behind HMSN-P, delaying disease onset and extending survival ...
A new review finds that the pathological protein TDP-43, the hallmark of ALS, accumulates in muscle, skin, glands and other ...
Seeds prepared from the spinal cords of ALS patients homozygous for the SOD1 D90A mutation transmitted two distinct strains of ...
A 26-year CDC WONDER analysis of 170,570 US deaths finds motor neuron disease death counts rose 47 percent while age-adjusted ...
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© 2025 Scienmag - Science Magazine