DRG uses cookies to improve your experience on this website. Some of the cookies we use are essential for parts of the website to operate. Please be aware that if you continue without changing your cookie settings, you consent to this. For more information on our use of cookies, please review our cookie policy.

Research & Reports

Searching in Biopharma (1620)

Spinal Muscular Atrophy | Landscape & Forecast | Disease Landscape & Forecast

Spinal Muscular Atrophy | Landscape & Forecast | Disease Landscape & Forecast

Thank you!

Your request has been received by DRG. A represantative will contact you shortly to provide more details on the research and data contained in this report and ensure that it will meet your current research needs.

Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by progressive muscle weakness due to the loss of anterior horn motor neurons. Nearly all cases of SMA are caused by homozygous deletion or mutation of the SMN1​ gene. SMA ranges widely in severity, and symptom onset usually occurs during childhood. In its most serious form, SMA is lethal in infancy. Although no treatments yet exist for SMA, several exciting therapies in late-phase development may enter the market in the near term, and several others are in earlier stages of clinical development. The bulk of the pipeline focuses on correcting the underlying defect in SMA—i.e., the deficient expression of the SMN protein.

  • Pub Date: June 2016
  • Author(s): Ian Love, PhD

Request report

Recent reports:
You may also be interested in: