Leukodystrophy Overview
Learn About Leukodystrophy
- Leukodystrophy
Ingeborg Mann-Krageloh practices in Tuebingen, Germany. Ms. Mann-Krageloh is rated as an Elite expert by MediFind in the treatment of Leukodystrophy. Her top areas of expertise are Metachromatic Leukodystrophy, Leukodystrophy, CACH Syndrome, Pontocerebellar Hypoplasia, and Bone Marrow Transplant.
Odile Tanguy-Boespflug practices in Paris, France. Ms. Tanguy-Boespflug is rated as an Elite expert by MediFind in the treatment of Leukodystrophy. Her top areas of expertise are Leukodystrophy, Spastic Paraplegia Type 2, CACH Syndrome, and Pelizaeus-Merzbacher Disease.
Childrens Health Care Associates Inc
Adeline Vanderver is a Pediatric Neurologist and a Neurologist in Philadelphia, Pennsylvania. Dr. Vanderver is rated as an Elite provider by MediFind in the treatment of Leukodystrophy. Her top areas of expertise are Aicardi-Goutieres Syndrome, Leukodystrophy, CACH Syndrome, Pol 3-Related Leukodystrophy, and Gastrostomy.
Summary: This study is a national, non-randomized, open-label, multi-site with minimal risk study in adult with adrenomyeloneuropathy (AMN), childhood and adult subjects with cerebral ALD (cALD), juvenile/adult metachromatic leukodystrophy (MLD) and adults with leukoencephalopathy and axonal spheroids and pigmented glia (ALSP). 49 subjects will be enrolled with one blood sample collection during one of the...
Summary: This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to transduce patient-derived hematopoietic stem cells (HSCs), with the goal of achieving therapeutic gene correction through transplantation of genetically modified HSCs. The primary objectives are to evaluate the safe...
Published Date: May 02, 2022
Published By: Genetic and Rare Diseases Informnation Center

