USANew treatments and approvals
First drug for balance problems in ataxia-telangiectasia, levacetylleucine, approved in the USA
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The US Food and Drug Administration (FDA) approved levacetylleucine for balance and walking problems in ataxia-telangiectasia (A-T). Children weighing at least 15 kilograms and adults can use it. The approval is based on a trial with 73 patients. Patients scored better while taking the drug. Common side effects were falls, skin cuts and urinary tract infection.
What does it mean for patients and families?
This drug only targets the balance problem. It does not change the risk of immune problems or cancer. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Ataxia-telangiectasia (A-T)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.