Join the Fight for Innovative Gene Therapy

Please join me for the cause for a cure to fund clinical trials.

It is rare to find a cure for rare and progressive disorders. But, UMass and St. Jude had a quick turnaround for one of the rarest metabolic disorders - Sialidosis through Neu1 gene therapy. The application of the same genetic modification is also found to open the path to treat Alzheimer’s, other neurodegenerative conditions, and a few cancers by correcting the metabolic pathways.

Patients with Sialidosis Type-2 have severe complications with deformities from birth.

Patients with Sialidosis Type-1 progress to lose their independence for daily living starting at pre-teen.

Unfortunately, families with one or more of their children suffer with the condition, as does mine.

Before it advances, we are working with NIH, St. Jude, and UMass to expedite the gene therapy for clinical trials for a 2-3 year turnaround. It takes $350k to serve approximately 10 patients. We need more human trials after the first clinical trials before it is released as a treatable drug.

These patients are heroes to try those clinical trials that not only treat Sialidosis but also pave a path forward for long-lasting Alzheimer’s and other neurodegenerative conditions with Neu1 gene therapy.

Why wait?? When research is available for the most long-lasting and debilitating conditions.

Help us to initiate clinical trials…

Join me in the cause

Please click on the “Donate for Sialidosis” button from www.curemucolipidosis.org

Please share and help the cause.

https://www.massachusetts.edu/news/bridge-grants-support-research-rare-sialidosis-disease-and-patch-prevent-pressure-ulcers

https://www.stjude.org/media-resources/news-releases/2013-medicine-science-news/gene-therapy-bolsters-enzyme-activity-to-combat-alzheimer-s-disease-in-mice.html