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Jun 21, 2024 · The study will examine effects on the neurons, mitochondria, neuroinflammation, nerve growth factors, health of the retina, and abnormal protein clumping. Behavioral outcomes of movement performance and anxiety-like behaviors will also be measured in the mice.
Jun 21, 2024 · Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is considered an orphan disease, which classifies it for special considerations in drug development and policy. It is...
Jun 21, 2024 · “This trial shows promise for improving the lived experience of not only the people diagnosed with Sanfilippo syndrome, but also their families who face countless disease-related stressors and heartache.” This study supports the potential of anakinra as a therapeutic option for Sanfilippo syndrome.
Jun 21, 2024 · Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is considered an orphan disease, which classifies it for special considerations in drug development and policy. It is...
Jun 24, 2024 · Sanfilippo syndrome is a fatal, childhood, neurodegenerative, disorder—with no approved treatment—that involves neuroinflammation among multiple pathologies.
Jun 12, 2024 · Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease with no approved treatment that primarily affects the CNS and is characterized by rapid neurodegeneration, with onset in early childhood.
Jun 28, 2024 · Fruit flies shed light on ultra-rare San... Tuesday, 2nd Apr 24. Four new projects for Sanfilippo syndrom...
