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.

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 ...

Bill Nye Shares Family's Ataxia Struggle: 'Every Time You Lose Your Balance, You Think This Is the End' (Exclusive) The beloved 'Science Guy' says many of his family members suffer from a rare ...

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 ...