
SMA-PME Research SMA-PME Research is an approved 501(c)(3) public charity that conducts research to find a cure for Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME) and ohter medical conditions directly related to finding a cure for SMA-PME. These illnesses are genetic and neurological in nature. SMA-PME is a lysosomal storage disorder caused by an acid ceramidase deficiency in the ASAH1 gene. Promising therapies that we have funded include but are not limited to Gene Modified Cell Therapy research led by Drs. Jeffrey Medin (Medical College of Wisconsin) and Michelle Allen-Sharpley (Cedars-Sinai). Patient Advocacy Organization

SMA-PME Research

SMA-PME Research is an approved 501(c)(3) public charity that conducts research to find a cure for Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME) and ohter medical conditions directly related to finding a cure for SMA-PME. These illnesses are genetic and neurological in nature. SMA-PME is a lysosomal storage disorder caused by an acid ceramidase deficiency in the ASAH1 gene. Promising therapies that we have funded include but are not limited to Gene Modified Cell Therapy research led by Drs. Jeffrey Medin (Medical College of Wisconsin) and Michelle Allen-Sharpley (Cedars-Sinai).

Patient Advocacy Organization