The US Food and Drug Administration (FDA) has rejected PTC Therapeutics’ new drug application (NDA) for vatiquinone in ...

FDA rejected PTC Therapeutics' vatiquinone for Friedreich's ataxia, citing insufficient efficacy data and requiring another ...

The U.S. Food and Drug Administration has declined to approve PTC Therapeutics' drug to treat a rare genetic disorder with ...

Ataxia telangiectasia (AT) is a rare, genetic condition that typically affects balance, coordination, the nervous system, the immune system, and the spine.

An OU researcher has made a breakthrough discovery while studying a genetic defect. The discovery could change how the condition is diagnosed and studied.

This media is in the public domain (free of copyright restrictions). You can copy, modify, and distribute this work without contacting the Smithsonian. For more information, visit the Smithsonian's ...

Localization and Network Connectivity of Lesions Causing Limb Ataxia in Patients With Stroke. Neurology, 2024; 103 (6) DOI: 10.1212/WNL.0000000000209803 ...

The U.S. Food and Drug Administration has denied approval for PTC Therapeutics' oral medication for a rare genetic disorder ...