# Neurogenetic and Muscular Disorders Research

**Type:** Topics  
**Canonical URL:** https://scholariq.org/topics/neurogenetic-and-muscular-disorders-research-2/

## Facts

| Field | Value |
| --- | --- |
| Citations | 469,170 |
| Description | This cluster of papers focuses on the gene therapy, molecular genetics, and clinical management of Spinal Muscular Atrophy (SMA). It covers topics such as SMN1 and SMN2 genes, Nusinersen treatment, antisense oligonucleotide therapies, motor neuron pathology, and the role of survival motor neuron protein in SMA. The cluster also discusses potential therapeutic strategies, including gene replacement therapy and the use of small molecules to modulate SMN expression. |
| Domain | Health Sciences |
| Field | Medicine |
| OpenAlex ID | https://openalex.org/T12400 |
| Works | 44,785 |

## Topic researchers

Showing 12 of 20.

- [Simon I Hay](https://scholariq.org/researchers/simon-i-hay/)
- [Theo Vos](https://scholariq.org/researchers/theo-vos/)
- [Ali H. Mokdad](https://scholariq.org/researchers/ali-h-mokdad/)
- [Mark J. Daly](https://scholariq.org/researchers/mark-j-daly/)
- [Stacey Gabriel](https://scholariq.org/researchers/stacey-gabriel/)
- [Valery L. Feigin](https://scholariq.org/researchers/valery-l-feigin/)
- [Matthias Mann](https://scholariq.org/researchers/matthias-mann/)
- [Gonçalo R. Abecasis](https://scholariq.org/researchers/goncalo-r-abecasis/)
- [Farshad Farzadfar](https://scholariq.org/researchers/farshad-farzadfar/)
- [Richard A. Gibbs](https://scholariq.org/researchers/richard-a-gibbs/)
- [John Q. Trojanowski](https://scholariq.org/researchers/john-q-trojanowski/)
- [Chris Sander](https://scholariq.org/researchers/chris-sander/)

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Source: ScholarIQ — public research metadata, principally OpenAlex. See https://scholariq.org/sources/ for provenance and https://scholariq.org/methodology/ for what these figures mean.