A new meta-analysis on the efficacy & safety of valproic acid (VPA) for Spinal Muscular Atrophy.
The study showed out:
Improvement of the gross motor functions of SMA patients. However, no improvements to other motor functions, such as the respiratory function.
In addition, VPA appears to be a relatively safe drug, although the treatment may be associated with a wide range of AEs (including body weight increase, fatigue, fever, flu-like symptoms, irritability and pain).
Valproic acid is often used in SMA type 1 and belongs to OFF Label Therapy. In addition, valproic acid is used in the treatment of epplepsy
Learn more and click this link to our colleagues from Axel Springer
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