Details

Autor(en) / Beteiligte

• Kim, Jeong-Ki
• Monani, Umrao R.

Titel

Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

Ist Teil von

• Neuron (Cambridge, Mass.), 2018-03, Vol.97 (5), p.1001-1003

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2018

Quelle

Elektronische Zeitschriftenbibliothek (Open access)

Beschreibungen/Notizen

• Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients. Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.