Spinal Muscular Atrophy (SMA) Stem Cell Medical Reports

Background and Objectives: SMA1 is a genetic disease causing progressive apoptosis of the second motoneuron, leading to complete paralysis. Given the reported efficacy of mesenchymal cells in treating other neurological

Major Points and Findings: Introduction to Stem Cells: Human embryonic stem cells (hESCs) are a valuable resource for studying human development and diseases. However, their use is limited due to

  Major Points: Efficacy: The study found significant improvements in CHOP INTEND and motor-milestone scores in SMA patients at both short- and long-term follow-ups. CHOP INTEND is a measure of

Introduction to SMA: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and is one of the leading genetic causes of infant mortality. The disease manifests as progressive muscle

  Purpose of Review SMA is an inherited neurodegenerative disorder that manifests in childhood. It is caused by a deficiency of the survival motor neuron (SMN) protein. This deficiency leads

Spinal Muscular Atrophy (SMA) Authors: Stephen J. Kolb, M.D., Ph.D., and John T. Kissel, M.D. Affiliation: Department of Neurology and Department of Biological Chemistry and Pharmacology, The Ohio State University