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  • Wiki Nusinersen (Spinraza) rel 0.50

    Spinal Muscular Atrophy ([spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy))[@fda]. Approved by the U.S. Food

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  • Spinal Muscular Atrophy (SMA) [spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy) is caused

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  • spinal-muscular-atrophy | spinal-cord - tdp-43 | tdp-43-proteinopathy

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  • Wiki risdiplam rel 0.28

    Spinal Muscular Atrophy Background ### Disease Pathophysiology [spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy

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  • spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy) (SMA). It delivers a functional copy

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  • spinal-muscular-atrophy (2022)( (2022)](https://doi.org/10.1007/s00115-021-01256-0) 6. [Unknown

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  • spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy). ## Prognosis NIID is a slowly progressive

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  • Wiki STMN2 (Stathmin-2) rel 0.10

    spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy) models, STMN2 expression is reduced, and restoring

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  • Wiki crispr-gene-editing rel 0.10

    spinal-muscular-atrophy SMN2 exon 7 inclusion batten-disease CLN gene

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  • Wiki tofersen rel 0.10

    spinal-muscular-atrophy](/diseases/spinal-muscular-atrophy), demonstrating broad applicability of ASO technology

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POST /api/scidex/rpc
{
  "verb": "scidex.search",
  "args": {
    "q": "",
    "limit": 20,
    "sort": "relevance"
  }
}