FDA approves first treatment for Rett syndrome

The Food and Drug Administration (FDA) of USA announced Monday that it has approved Daybue (trofinetide) oral solution as the first treatment for Rett syndrome, a rare genetic neurological disorder.

The approval of the US regulator authorizes its use in adults and children from two years of age.

Those affected can ingest Daybue orally or through a gastric tube, twice a day, in the morning and at night, and the recommended dose depends on the person’s weight, the FDA said in its statement.

Rett syndrome is considered a rare genetic neurological disorder that affects the way the brain develops.

Most affected babies grow normally up to six months, but begin to lose faculties from then on.

The FDA noted that it primarily affects women (one in 10,000) and rarely men. The Rett Syndrome Research Trust, which promotes research to achieve its cure, estimates that there are about 15,000 people affected in the United States and 350,000 worldwide.

The Spanish association dedicated to this disease specifies on its website that it leads to a serious disability that alters almost all aspects of life: from the ability to speak, walk and eat to even breathing normally. Its hallmark is the Constant repetitive movements of the hands.

The FDA noted that there are no contraindications to taking Daybue and stressed that possible side effects include diarrhea and weight loss. Less common included cases of fever, anxiety, and tiredness.

Source: EFE.