New Expanded Access Trials For FENIB and LOTS Patients Set to Begin

According to a story from, the corporation Retrotope recently announced the initiation of single-patient, expanded access trials of its experimental product RT001. The patients involved have familial encephalopathy with neuroserpin inclusion bodies (FENIB) and late onset Tay-Sachs disease (LOTS).

RT001 possesses a unique mechanism of action that allows it to protect against damage caused by free radicals. Damage from free radicals can result in premature cell death and frequently occurs in neurodegenerative diseases.

FENIB is a very rare progressive disorder that affects the nervous system. It is characterized by seizures and dementia primarily. Symptoms often first begin with difficulties of concentration and attention. Eventually, FENIB patients lose their ability to function in day to day life and require round-the-clock care. It caused by genetic mutations. Symptoms can appear as early as the late teens, but less severe cases appear in the forties or fifties. To learn more about FENIB, click here.

Late onset Tay-Sachs disease (LOTS) is distinguished from typical Tay-Sachs because it first appears in adulthood and is generally less severe. Typically, Tay-Sachs disease appears in the first few months of life and causes death in early childhood. It is a genetic disorder that causes the death of nerve cells in the spinal cord and brain. LOTS symptoms first appear in the patient’s thirties or forties, but can also occur sooner. Unlike the more common forms, it is not lethal. This because the progression of the disease eventually stops on its own. Symptoms include unsteady gait, loss of walking ability, difficulty speaking and swallowing, psychosis, cognitive decline, and spasticity. To learn more about LOTS, click here.

LOTS shares several symptoms with other diseases such as Friedreich’s ataxia and infantile neuroaxonal dystrophy (INAD); RT001 is also being tested against these diseases. Trials have demonstrated that RT001 has been effective in treating these diseases, successfully halting progression and even slightly improving symptoms. It was these results that persuaded Dr. Peter Milner, who is the Chief Medical Officer at Retrotrope, that the treatment could be effective in LOTS and FENIB as well.

Hopefully, these new trials will indicate that RT001 is capable of treating a variety of neurodegenerative disorders.

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