Raffi Kodikian Discusses the Promising New Orphan Drug Treatment for Rett Syndrome

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Raffi Kodikian Discusses the Promising New Orphan Drug Treatment for Rett Syndrome


After his niece was diagnosed with a rare genetic condition known as Rett syndrome, Raffi Kodikian began dedicating both time and money to helping support the organizations that are pushing toward a cure. Organizations like Rett Syndrome Research Trust (RSRT) have spent years trying to help find a cure for a condition that had been considered incurable. Now, a company called Taysha Gene Therapies, Inc may finally be on the verge of releasing a drug that is capable of significantly curbing the symptoms that Kodikian’s niece, along with 1 in every 10,000 young girls across the world, experiences.

What is Rett Syndrome?

Rett syndrome is a rare genetic neurological disorder that is found almost exclusively in women. Constant, repetitive hand movements is the hallmark symptom of Rett syndrome, though other symptoms can include loss of speech, loss of muscle tone, scoliosis, breathing issues, sleep disturbances, seizures, and a slowed rate of growth for the patient’s head, hands, and feet.

Rett syndrome occurs in about 1 out of every 10,000 female births, and the symptoms typically show up around the age of 6 to 18 months. Since Rett syndrome was first cataloged in medical literature in the 1960s, no cure for the condition has been found. Thanks to the hard work of Taysha Gene Therapies, Inc., though, this could be about to change.

TSHA-102 – The Most Promising Rett Syndrome Treatment to Date

According to Kodikian, TSHA-102 – a drug that has recently been given orphan drug designation by the FDA – marks the most significant breakthrough in the fight for a Rett syndrome cure since his niece was diagnosed with the condition. TSHA-102 works by regulating the MECP2 gene responsible for mutating and causing Rett syndrome on a cell-by-cell basis. In clinical trials, this drug has been shown to dramatically reduce Rett syndrome symptoms. Now, Kodikian is hopeful that TSHA-102’s orphan drug designation will help fast-track the drug to market.

Orphan drug designation is given to promising treatments for rare conditions that wouldn’t be profitable without government subsidies due to the small number of people afflicted with the conditions that they treat. According to Kodikian, this designation means that Taysha Gene Therapies, Inc. will be able to push forward with the development and release of TSHA-102 with the confidence that the company will be rewarded for its efforts.

Almost sixty years since Rett syndrome was first documented have gone by with almost zero progress toward a meaningful treatment, much less a potential cure. Now, Kodikian says that finally seeing some light at the end of the tunnel is something that has reinvigorated his passion for the cause. Only time will tell if TSHA-102 ends up as an effective treatment for Rett syndrome patients. To Kodikian, though, its orphan drug designation and clinical trial results are, at the very least, a highly promising start.