There are over 40 different lysosomal storage diseases, including Gaucher, Fabry, Pompe, Tay Sachs, Canavan, Sanfilippo Diseases, SP-1 Lyase Deficiency and many, many more. They each derive from mutations to the genetic code of an enzyme that is critical for normal cellular function, unique to each disease. These are progressive, debilitating and sometimes fatal diseases, each requiring a unique solution. Enzyme replacement therapy or Substrate Replacement Therapy are the most common approaches, but are generally not sufficient to address all of the disease manifestations.
