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

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

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

As part of the campaign, Nye has a subsite on the NAF website that educates people on the disorder, asking them to sign up with their details to become a Bill Nye Ataxia Advocate. It comes with ...

Just over the last two, three years, the FDA approved Skyclarys, which is the first drug, actually, to be approved for ataxia in general, specifically. It provides modest help, not a cure for the ...

III. Study results After 48 weeks, the L-arginine group showed an improvement of 0.96 ± 0.55 points in mean SARA scores, indicating a mild reduction in ataxia symptoms. In contrast, the placebo ...

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 declined to approve PTC Therapeutics' drug to treat a rare genetic disorder with ...