Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape

Emerging Gene Therapy Approaches in the Management of Spinal Muscular Atrophy (SMA): An Overview of Clinical Trials and Patent Landscape

Spinal muscular atrophy (SMA) is a rare autosomal recessive neuromuscular disease that is characterized by progressive muscle atrophy (degeneration), including skeletal muscles in charge of the ability to move. SMA is caused by defects in the SMN1 gene (Survival of Motor Neuron 1) which encodes a pr...

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Bibliographic Details
Main Authors: Aleksei S. Ponomarev, Daria S. Chulpanova, Lina M. Yanygina, Valeriya V. Solovyeva, Albert A. Rizvanov
Format: Article
Language:English
Published: MDPI AG 2023-09-01
Series:International Journal of Molecular Sciences
Subjects:
spinal muscular atrophy
SMN
gene therapy
clinical trials
patent landscape
Online Access:https://www.mdpi.com/1422-0067/24/18/13743

Internet

https://www.mdpi.com/1422-0067/24/18/13743

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