Stefania Corti practices in Milan, Italy. Corti is rated as a Distinguished expert by MediFind in the treatment of Infantile Axonal Neuropathy. She is also highly rated in 30 other conditions, according to our data. Her top areas of expertise are Primary Lateral Sclerosis, Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type 1, Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's Disease), and Bone Marrow Transplant.
Giacomo Comi practices in Milano, Italy. Comi is rated as a Distinguished expert by MediFind in the treatment of Infantile Axonal Neuropathy. He is also highly rated in 42 other conditions, according to our data. His top areas of expertise are Becker Muscular Dystrophy, Duchenne Muscular Dystrophy, Primary Lateral Sclerosis, Spinal Muscular Atrophy (SMA), and Bone Marrow Transplant.
Nereo Bresolin practices in Milan, Italy. Bresolin is rated as a Distinguished expert by MediFind in the treatment of Infantile Axonal Neuropathy. He is also highly rated in 37 other conditions, according to our data. His top areas of expertise are Primary Lateral Sclerosis, Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type 1, Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's Disease), and Bone Marrow Transplant.
Summary: This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
Summary: The purpose of this study is to gather information on the possible symptoms that patients with atypical neuronal ceroid lipofuscinosis type 2 (also known as aTPP1 or atypical tripeptidyl peptidase deficiency) have and how they change over time.
Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center