Daniel Grinberg practices in Barcelona, Spain. Grinberg is rated as an Elite expert by MediFind in the treatment of C Syndrome. He is also highly rated in 23 other conditions, according to our data. His top areas of expertise are C Syndrome, Mucopolysaccharidosis Type 3 (MPS III, Sanfilippo Syndrome), Hereditary Multiple Osteochondromas, and Osteochondroma.
Roser Urreizti practices in Barcelona, Spain. Urreizti is rated as an Elite expert by MediFind in the treatment of C Syndrome. She is also highly rated in 6 other conditions, according to our data. Her top areas of expertise are C Syndrome, Osteoporosis, Lambdoid Synostosis, and Metopic Ridge.
Susana Balcells practices in Barcelona, Spain. Balcells is rated as an Elite expert by MediFind in the treatment of C Syndrome. They are also highly rated in 9 other conditions, according to our data. Their top areas of expertise are C Syndrome, Hereditary Multiple Osteochondromas, Osteochondroma, and Osteoporosis.
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body. There is no cure for NPC, and symptoms only get worse over time. Symptoms can include seizures, difficulty moving or talking, or dementia. But symptoms can vary among different people with the disease. Some may have seizures, while ot...
Summary: This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic...
Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center