The Current Status of Spinal Muscular Atrophy Therapeutics

Authors James P. Van Meerbeke and Charlotte J. Sumner published a comprehensive article Progress and Promise: The Current Status of Spinal Muscular Atrophy Therapeutics on all of the therapeutics currently being researched for the treatment of Spinal Muscular Atrophy (SMA).


The article provides a fairly detailed explanation on how various treatment strategies are supposed to work. Furthermore it details in what phases of the drug pipeline all the identified components that might help in treating SMA are.


Considering the fact that the SMA causing gene had only been discovered in 1995, which triggered some rapid advances in the are, it can really be concluded that there is great progress and promise in the current development.

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