Help us stop
Lafora Disease

HELP US
STOP
TIME

LAFORA DISEASE

UNITED AGAINST LAFORA

Lafora disease is part of the Progressive Myoclonic Epilepsies group and is considered the most severe form of epilepsy.

It is a degenerative neurological disorder characterized by epileptic seizures, myoclonus, and cognitive decline.

It is caused by the malfunctioning of two proteins, Laforin and Malin. This malfunction is due to genetic alterations in one of the two genes, both located on chromosome 6: EPM2A and EPM2B.

This situation creates an error in glycogen metabolism, leading to its progressive accumulation in the brain.

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FOCUS ON

ION283
Recognition of a Historic Milestone in Lafora Disease Research

Today, with the ION283 ready to enter Phase 1 clinical trials, we can finally say that we are one step closer to realizing a dream that has accompanied us for years.

A DISEASE
THAT MAKES ITSELF KNOWN

The progression of the disease is devastating. Although a child is born with this disease, it typically does not manifest until adolescence. Children grow up normally, without noticeable problems, until they begin to experience seizures.

This is one of the cruelest aspects of Lafora. A normal, beautiful, and seemingly healthy teenager who has everything to live for is faced with nothing less than a “death sentence.”

The therapy is mainly palliative and aims to reduce the seizures.

There is currently no therapy that can slow down or stop the progression of the disease.

THE STORY OF GIORGIO

GIORGIO IS ONLY 16 YEARS OLD

Giorgio is a 16-year-old boy, and for him and his family, there is no more time.

In September 2023, Giorgio was diagnosed with a rare degenerative neurological disease, Lafora. While he was preparing to face life’s challenges head-on, this disease stopped him in his tracks. And by stopping him, it turned his family’s life upside down.

For Giorgio and his family, there is no more time!

Discover the story

OUR GOAL

SUPPORT
THE RESEARCH

We need your help to raise the necessary funds to initiate the Phase 1/2 clinical trial (safety and efficacy) of a new ASO drug specifically created for Lafora disease.

1.5 MILLION DOLLARS.
10 PATIENTS.
2 YEARS OF TREATMENT.

The clinical trial requires two years during which families will need to bring their children to Dallas every three months. In addition to travel and accommodation costs, parents will have to cover the cost of the trial itself, which has been estimated at $150,000 per patient for two years of treatment.

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