Sentences with phrase «as mucopolysaccharidosis»
Jack Baird, who is four years old and from Sunderland, has the condition Sanfilippo Disease, also known as Mucopolysaccharidosis (MPS) III, which affects around one in 85,000 people in the UK.
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Clinical research activities are focused on the implementation of experimental protocols based on gene or cell therapy for the treatment of genetic blood disorders such as ADA - SCID, Wiskott - Aldrich Syndrome, Metachromatic Leukodystrophy, Beta - thalassemia, Chronic granulomatous disease, Mucopolysaccharidosis Type I (Hurler).
Lysosomal storage diseases such as Niemann - Pick disease, mucopolysaccharidosis and gangliosidosis (GM1) have been described in Siamese cats, as has systemic amyloidosis.