We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Novel Therapies.
- Abstract
The article presents multiple opinions on novel therapies of lysosomal storage diseases (LSDs). Kay MacDermot's commentaries concern the use of bicyclic derivatives of L-idonojirimycin for certain forms of Gaucher disease (GD). MacDermot also expresses his opinion on a gene therapy to the diaphragm for respiratory dysfunction in Pompe disease. There are also comments by Atul Mehta on a retrospective study on Miglustat therapy in type 1 Gaucher's disease.
- Subjects
LYSOSOMAL storage diseases; THERAPEUTICS; GAUCHER'S disease treatment; GENE therapy; RESPIRATORY insufficiency; GLYCOGEN storage disease type II
- Publication
Current Medical Literature: Lysosomal Storage Disease, 2013, Vol 11, Issue 4, p122
- ISSN
1473-0901
- Publication type
Article