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Literature DB >> 32206168

Spinal Muscular Atrophy: Huge Steps.

Kelly Howell, Rebecca M Gibbs, Lee L Rubin.

Abstract

Spinal muscular atrophy is the number one genetic cause of infant death. Until recently, half the babies born with it would die before their second birthdays, their hearts and lungs becoming too weak to continue. Medical care improved the odds somewhat, but new discoveries and therapeutic developments have improved survival rates significantly-and more good news may be on the horizon. Copyright 2019 The Dana Foundation All Rights Reserved.

Entities: Disease Species

Year: 2019 PMID： 32206168 PMCID： PMC7075499

Source DB: PubMed Journal: Cerebrum ISSN： 1524-6205

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References

29 in total

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Spinal Muscular Atrophy: Huge Steps.

1. Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy.

2. Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member.

Review 3. Spinal muscular atrophy: going beyond the motor neuron.

4. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice.

5. Observational study of spinal muscular atrophy type I and implications for clinical trials.

6. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Review 7. The role of survival motor neuron protein (SMN) in protein homeostasis.

8. Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.

9. Blocking extracellular activation of myostatin as a strategy for treating muscle wasting.

10. CK-2127107 amplifies skeletal muscle response to nerve activation in humans.