[0004] Mucopolysaccharidosis type III (MPS III) or Sanfilippo disease is a lysosomal storage disease, of the mucopolysaccharidosis group, characterized by severe and rapid intellectual degradation.
The first candidate medication, LYS-SAF302, for the treatment of mucopolysaccharidosis IIIA (MPS IIIA) or Sanfilippo syndrome type A, is in late-stage clinical development (phase III anticipated to begin in early 2018);
The first candidate medication, LYS-SAF302, for the treatment of mucopolysaccharidosis IIIA (MPS IIIA) or Sanfilippo syndrome type A, is in late-stage clinical development (phase III anticipated to begin in early 2018);
On July 13, 2017, the journal Lancet Neurology published the results of a gene therapy trial conducted in four children with Sanfilippo type B syndrome (also known as MPS IIIB).
On July 13, 2017, the journal Lancet Neurology published the results of a gene therapy trial conducted in four children with Sanfilippo type B syndrome (also known as MPS IIIB).
On July 13, 2017, the journal Lancet Neurology published the results of a gene therapy trial conducted in four children with Sanfilippo type B syndrome (also known as MPS IIIB).
There are four types of Sanfilippo syndrome, each categorized according to the type of enzyme missing or deficient.
Sanfilippo syndrome causes numerous deaths among children under the age of 10.
Sanfilippo syndrome is an incurable genetic disease and current treatments only address the symptoms rather than the causes.
pivotal trials of LYS-SAF302 conducted in Europe and the United States for Sanfilippo syndrome type A;
A child suffering from Sanfilippo Syndrome displays no anomaly at birth, the first symptoms appearing between 2 and 6 years of age.
My husband and I had never heard of Sanfilippo syndrome before the doctors told us in July 2005 that our dear and beautiful Ornella was suffering from the disease.
Andrew A. Sanfilippo, a partner at O’Donnell, Robertson & Sanfilippo LLP, is appointed a judge of the Superior Court of Justice in Toronto.
Andrew A. Sanfilippo, a partner at O’Donnell, Robertson & Sanfilippo LLP, is appointed a judge of the Superior Court of Justice in Toronto.
Andrew A. Sanfilippo, a partner at O’Donnell, Robertson & Sanfilippo LLP, is appointed a judge of the Superior Court of Justice in Toronto.
The present invention provides biomarkers for efficient and accurate characterization of Sanfilippo syndrome.
Author of Article: Hans Rollmann and Matteo Sanfilippo
In particular, the present invention provides biomarkers differentially expressed in Sanfilippo syndrome.
What are the best treatments for Sanfilippo Syndrome?
What are the latest advances in Sanfilippo Syndrome?
There are four different enzyme deficiencies that have been found to cause Sanfilippo syndrome.
Currently, there is no available treatment for Sanfilippo syndrome.
There is currently no specific treatment for Sanfilippo syndrome.
Anchoas Sanfilippo in Salting, The Petite 4-piece large
There is currently no cure for Sanfilippo syndrome.
There currently is not a treatment for Sanfilippo Syndrome.
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