FDA Gives Rare Pediatric Disease Designation to Experimental Drug for Treating Rett Syndrome

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A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a long time, meaning that those who have Rett syndrome have had no way to combat this symptom. Now the FDA has awarded Rare Pediatric Disease designation to sarizotan, a drug meant to specifically treat the apnea that is associated with Rett syndrome. Newron, the company that is currently developing this drug, is excited for the results of its study, Sarizotan Treatment of Apneas in Rett Syndrome (STARS), in order to fully evaluate the drug and its future.

About Rett Syndrome

Rett syndrome is a severe, rare neurological disorder that is associated with a mutation on the X chromosome. Despite the fact that it is a genetic disease, it is rare that it is inherited. A random mutation is often the cause. Due to its correlation with the X chromosome, it mainly affects females. Males who have this condition rarely live past birth. It is estimated that one in every 10,000 females have Rett syndrome. Symptoms tend to appear in infants aged 12 to 18 months. These symptoms can include slow brain growth, which results in a smaller head, issues with hand movement and muscle coordination, no language skills, social anxiety, apnea, seizures, and a tense, irritable disposition. The disorder is characterized by the deterioration of fine and gross motor skills, which leads to neurological, cognitive, and autonomic issues. Those with Rett syndrome also have a shorter life span, which varies from person to person based on the onset and severity of symptoms. After noticing symptoms and a clinical examination, doctors must rule out other conditions, such as autism. There is no current cure for this disorder, and treatment is directed towards symptoms, such as anti-seizure drugs. Physical, occupational, and behavioral therapy are methods of treatment as well.

About Sarizotan and STARS

The study, Sarizotan Treatment of Apneas in Rett Syndrome (STARS), was to evaluate the safety, tolerability, and efficacy of the drug in order to ensure that it was ready for patients. Sarizotan is meant to combat the respiratory symptoms of Rett syndrome, which can affect the entire body. If the respiratory system is not functioning correctly it can also cause issues with biochemistry, emotions, circulation, digestion, and musculoskeletal structures. The drug also received Orphan Drug designation.

About Newron

Newron is a biopharmaceutical company that focuses on creating therapies for disease of the peripheral and central nervous system; it is based out of Italy. In the past it has worked on therapies for the treatment of Parkinson’s disease, which are now available in countries across the world. Currently it is working on treatments for Rett syndrome, schizophrenia, and rare pain indications.

Find the original article here.

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