
The New Hope Research Foundation is a non-profit organization dedicated to:
– Helping find a genetic cure for GM2 gangliosidosis and other lysosomal storage diseases that affect the central nervous system, and
– Providing hope to patients and their families currently facing the degenerative and debilitating aspects of these diseases.
To accomplish these goals, the New Hope Research Foundation has been established to coordinate, conduct, and financially support:
– Research on gene therapy and the means for infusing or delivering gene vectors to correct lysosomal enzyme deficiencies of the central nervous system
– Clinical studies intended to show safety and efficacy of gene therapy for the central nervous system aspects of lysosomal storage diseases,
– Regulatory applications required to initiate investigations of clinical studies and the regulatory submissions required to allow patient access to proven therapies, and
– Education and communication for patients and families on the clinical manifestations and molecular basis of gangliosidosis and related diseases, on the mechanism of gene therapy, and on the status of related research and clinical studies.