Sandrine Passemard practices in Paris, France. Passemard is rated as an Elite expert by MediFind in the treatment of Achalasia Microcephaly Syndrome. She is also highly rated in 9 other conditions, according to our data. Her top areas of expertise are Achalasia Microcephaly Syndrome, Microcephaly, Cortical Dysplasia, and Microcephalic Osteodysplastic Primordial Dwarfism Type 2 (MOPD2).
Alain Verloes practices in Paris, France. Verloes is rated as an Elite expert by MediFind in the treatment of Achalasia Microcephaly Syndrome. He is also highly rated in 66 other conditions, according to our data. His top areas of expertise are Achalasia Microcephaly Syndrome, Microcephaly, Noonan Syndrome, and RASopathies.
Laurence Faivre practices in Dijon, France. Faivre is rated as an Elite expert by MediFind in the treatment of Achalasia Microcephaly Syndrome. He is also highly rated in 116 other conditions, according to our data. His top areas of expertise are Cohen Syndrome, Microcephaly, Achalasia Microcephaly Syndrome, and Hypotonia.
Summary: MELPIDA is proposed for the treatment of subjects with SPG50 and targets neuronal cells to deliver a fully functional human AP4M1 cDNA copy via intrathecal injection to counter the associated neuronal loss. Outcomes will evaluate the safety and tolerability of a single dose of MELPIDA, which will be measured by the treatment-associated adverse events (AEs) and serious adverse events (SAEs). Second...
Summary: The goal of this registry is to collect information on individuals with Microcephalic Osteodysplastic Primordial Dwarfism Type II (also called MOPDII) and other forms of microcephalic primordial dwarfism. The study team hopes to learn more about these conditions and improve the care of people with it by establishing this registry.