Brain changes in severe forms of spinal muscular atrophy (SMA) were progressive over time possibly due to severe reduction in survival motor neuron (SMN) protein levels.
Why this matters
Most patients with SMA type 0 die in the first weeks of life; early intervention with SMN2-splicing modulators or gene transfer of SMN1 might interfere with or modify progression.
Want to read more?
Log in or sign up to access all Neurodiem content.
Already have an account? Log In
International Medical Press is a global provider of independent medical education. Its mission is to provide healthcare professionals with high-quality, trusted medical information with the aim of helping optimize patient care.
No responsibility is assumed by International Medical Press for any injury and/or damage to persons or property through negligence or otherwise, or from any use or operation of any methods, products, instructions, or ideas contained in the material herein. Because of rapid advances in the medical sciences, International Medical Press recommends that independent verification of diagnoses and drug dosages should be made. The opinions expressed do not reflect those of International Medical Press or the sponsor. International Medical Press assumes no liability for any material contained herein.