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In lysosomal storage diseases that affect other organs such as liver or muscle, doctors treat patients with repeated infusions of the missing enzyme into blood vessels, but infusion into vessels will not work when brain tissue is involved, as in Batten disease.
These prominent researchers share Vtesse's mission to develop new treatments to significantly improve the quality of life for people suffering from NPC and other lysosomal storage diseases (LSDs)," said Ben Machielse, Drs, President and Chief Executive Officer of Vtesse, Inc.
They specialty enzymes based drugs are also used for treating hay fever, lysosomal storage diseases and severe joint pain caused by age.
These diseases include Tuberous Sclerosis Complex, PEComas and hemangiomas (project 2), metabolic diseases (projects 3), lysosomal storage diseases (project 4).
MPS I is one of the most common lysosomal storage diseases to affect the central nervous system, which in severe form can become Hurler syndrome.
Lysosomal storage diseases which are characterized by non-immune hydrops fetalis include GM1-gangliosidosis, mucopolysaccharidosis type VII, gaucher disease, sialidosis and Salla disease (8).
Health professionals in the field of Lysosomal Storage Diseases (LSD), a collective term for over 50 rare inherited metabolic diseases that mostly affect children, from across the globe attended a conference organised by the Hamad Medical Corporation to discuss the recent advances in basic and clinical aspects of the diseases.
Insights into the diagnosis and treatment of lysosomal storage diseases.
London, May 10 (ANI): Scientists at The Scripps Research Institute have shed light on a mechanism that enables a potential treatment for Gaucher's disease and other lysosomal storage diseases.
Tifft, MD, PhD, FAAP, FACMG {CT}: In order to describe gangliosidoses, you really need to describe what a lysosomal storage disease is, because gangliosidoses are one of approximately 40 to 50 different lysosomal storage diseases.
uk - The Society for MPS represents 1,200 children and adults in the UK suffering from all lysosomal storage diseases.