Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases.

@article{Hemsley2009DeliveryOR,
  title={Delivery of recombinant proteins via the cerebrospinal fluid as a therapy option for neurodegenerative lysosomal storage diseases.},
  author={Kim M Hemsley and John J. Hopwood},
  journal={International journal of clinical pharmacology and therapeutics},
  year={2009},
  volume={47 Suppl 1},
  pages={S118-23}
}
Patients with lysosomal storage diseases (LSDs) have a greatly diminished lifespan and reduced quality of life, particularly those with neurological manifestations. There are few therapeutic options available to treat the neurological signs and symptoms of LSDs. It is, therefore, imperative that efficacious and tolerable treatments are developed. Hematopoietic stem cell transplantation is carried out in some LSDs in which there is neurological involvement. However, this approach is associated… CONTINUE READING

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