Dr Whitley is a key opinion leader in the field of lysosomal diseases
, founding Course director for WORLDSymposium, and Principal Investigator for the global Lysosomal Disease
Infantile neuronal ceroid lipofuscinosis (INCL) is a severe lysosomal disease
caused by mutations in the CLN1 gene, which encodes the soluble lysosomal enzyme Palmitoyl- Protein-Thioesterase-1 (PPT1) and result in osmiophilic granules accumulating in lysosomes and leading to neuroinflammation, neurodegeneration and death.
Hunter syndrome (mucopolysaccharidosis type II (MPS II)) is a lysosomal disease
characterized by deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S).
[G.sub.M1]-gagliosidosis is a relatively rare lysosomal disease
, presenting clinically with progressive neurological deterioration mainly in infancy and childhood with various somatic manifestations.
Citation: "Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease
"; Ana Sergijenko et al.; Molecular Therapy, 2013; DOI: 10.1038/mt.2013.141
Genzyme said on Friday that it has presented positive new data from its Phase 3 ENGAGE and ENCORE studies of its investigational oral therapy eliglustat tartrate for Gaucher disease type 1 at the 9th Annual Lysosomal Disease
Network WORLD Symposium in Orlando, Fla.
In February 2010, the company announced Phase III data at the Lysosomal Disease
Network's WORLD symposium.
[Otospongiosis, a cellular and enzymatic lysosomal disease
. Cyto-clinical correlation].
M2 PHARMA-February 10, 2012-Synageva BioPharma presents positive interim data at eighth annual Lysosomal Disease
Network (LDN) World Symposium(C)2012 M2 COMMUNICATIONS
Biopharmaceutical company Amicus Therapeutics (Nasdaq:FOLD) said on Thursday that the preclinical studies of its AT3375 for Gaucher disease were presented at the eighth Annual Lysosomal Disease
Network WORLD Symposium (LDN WORLD).
Protalix BioTherapeutics Inc (NYSE:PLX) (AMEX:PLX) (TASE:PLX) announced on Thursday that new clinical data on taliglucerase alfa will be presented at the 8th Annual Meeting of the Lysosomal Disease
Network: WORLD Symposium 2012 being held on 8-10 February in San Diego.
These two-year results were presented at the Lysosomal Disease
Network WORLD Symposium earlier this year, Genzyme added.