Smart4fabry / Communication  / Newsletter #6

Newsletter #6

Lysosomal storage disorders (LSDs) are inherited rare diseases caused by the lack or malfunction of proteins involved in lysosomal activity. Enzyme replacement therapies (ERTs, based on the administration of a functional version of the defective enzyme) have gained clinical relevance in LSD health care. Therefore, production of therapeutic enzymes is a key aspect in the development of therapies for many rare diseases.


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