View Main Condition: Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome)
Valeria De Pasquale practices in Naples, Italy. De Pasquale is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B). She is also highly rated in 3 other conditions, according to our data. Her top areas of expertise are Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B), Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome), Mucopolysaccharidoses (MPS), and Multiple Sulfatase Deficiency.
Kim Mcbride is a Medical Genetics specialist and a Pediatrics expert in Columbus, Ohio. Mcbride has been practicing medicine for over 36 years and is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B). He is also highly rated in 14 other conditions, according to our data. His top areas of expertise are Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome), Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B), Mucopolysaccharidoses (MPS), and Mucopolysaccharidosis Type 3A (MPS IIIA, Sanfilippo Syndrome A). He is licensed to treat patients in Ohio. Mcbride is currently accepting new patients.
Patricia Dickson is a Pediatrics specialist and a Medical Genetics expert in Saint Louis, Missouri. Dickson has been practicing medicine for over 24 years and is rated as an Elite expert by MediFind in the treatment of Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B). She is also highly rated in 5 other conditions, according to our data. Her top areas of expertise are Mucopolysaccharidosis Type 1 (MPS I, Hurler Syndrome), Mucopolysaccharidoses (MPS), Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome), Mucopolysaccharidosis Type 3B (MPS IIIB, Sanfilippo Syndrome B), and Hip Replacement. She is licensed to treat patients in Missouri. Dickson is currently accepting new patients.
Summary: The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).
Summary: This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervent...
Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center