Parents fighting to cure LNPK

There have been 20 other published cases of LNPK globally, with Olive being the first in the United States. With less than 30 known cases, LNPK is currently considered an ultra-rare disease. Through advances in the accessibility to genetic testing, this number will continue to increase.

Although our particular disease is ultra-rare, there are approximately 7,000 rare diseases affecting between 25 and 30 million Americans. Two-thirds of rare diseases impact children, and 30% will die before age 5. While these diseases are unique in their nature, 1 in 10 Americans are impacted. This is a problem worth solving.

We believe that every life has value, and refute the notion that medical treatments for rare diseases are less important than others. Although it may not be profitable for drug companies to develop individual cures for rare diseases, these lives are worth saving.

Our children deserve better.

Scientists are using gene therapy to treat diseases in three major ways: to replace missing or mutated genes, to add genes to the body, or to “turning off” genes that are causing problems.

To insert new genes directly into cells, scientists use a vehicle called a “vector.” Viruses are currently the most commonly used vectors in gene therapies because they have a natural ability to deliver genetic material into cells.

Before a virus can be used to carry therapeutic genes into human cells, it is modified to remove its ability to cause infectious disease.

There are over 300 gene therapies currently in clinical trials and 20 fully approved by the FDA.

Source: https://www.hopeforpdcd.org/

100% of all donations fund the research, treatment, and ultimately, finding a cure for LNPK. We have awarded a research grant to The Gray Lab at UTSW is to conduct a proof of concept study in mice models which will cost $400,000 over 3 years. All donations go directly to this research.

Yes. Olive is currently the only known LNPK patient in the United States, but it is very likely that other children will be diagnosed soon.

As genetic testing becomes more widely available and awareness levels rise, the research we are funding will forge a treatment path for undiagnosed and future-diagnosed LNPK children.

We are committed to unlocking the therapies for LNPK that will be used to treat other children for significantly less cost once the first clinical trial begins.

Because any given rare disorder affects so few patients, companies are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market.

Without meaningful support from the biotech industry or US government, it is becoming more and more common for rare disease families to join forces and raise the money required for cures for their children. Batten’s disease, SPG50, and SMARD have all achieved gene therapy treatments because these families moved mountains in honor of their children.

Source: https://www.hopeforpdcd.org/