A newly designed backpack, incorporating aerospace technology, shows promise as a balance aid for patients with the movement disorder ataxia. Research conducted by Radboud university medical center, ...

Please provide your email address to receive an email when new articles are posted on . Vatiquinone inhibits 15-Lipoxygenase, which regulates the pathways that Friedreich’s ataxia disrupts. PTC ...

The FDA rejected PTC Therapeutics' NDA for vatiquinone, citing insufficient efficacy evidence and requiring an additional study for resubmission. The MOVE-FA trial, a Phase II/III study, failed to ...

PTC Therapeutics has long been trying to workshop its vatiquinone into a marketable treatment for Friedreich's ataxia (FA) after a phase 3 fail in 2023. Despite its efforts, the FDA has snubbed the ...

The U.S. Food and Drug Administration has turned away PTC Therapeutics' proposed vatiquinone treatment for the rare, inherited neurodegenerative disorder Friedreich's ataxia over concerns about the ...

The FDA requires more evidence of efficacy for vatiquinone, a 15-lipoxygenase inhibitor, before approval for Friedreich ataxia treatment. Vatiquinone showed improvement in bulbar and upright stability ...

Apraxia is a neurological disorder that affects the mental processes involved in planning, task performance, and more. Ataxia is a neurological sign affecting muscle coordination and movement.

Immune checkpoint inhibitors (ICIs) have transformed the management of advanced and recurrent endometrial carcinoma (EC). ICI therapy can be associated with complex, multisystemic immune-related ...

Cerebellar ataxias represent a wide group of neurological diseases secondary to dysfunctions of cerebellum or its associated pathways, rarely coursing with acute-onset acquired etiologies and chronic ...

Armed with base editors, scientists from the laboratory of David Liu, PhD, professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute, and their ...

Quince Therapeutics is advancing eDSP for Ataxia-Telangiectasia, leveraging strong prior data in the 6-9 age group and an innovative AIDE platform. Recent insider buying, new analyst coverage with ...

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a clinical-stage nonprofit biotechnology company developing genetic medicines for ultra-rare and rare conditions, today announced that the ...