HERCULES: Development of an innovative muscle-directed gene therapy platform for lysosomal storage disorders Vrije Universiteit Brussel
muscle disorders that is caused by a genetic defect in acid
glucosidase (GAA). Typically, the afflicted patients gradually lose all
muscle function and ultimately die from heart and lung failure.
Current treatment with recombinant proteins alleviates some of the
symptoms but are not curative and largely inadequate since the large
majority of ...