In the CRL, the Agency concluded that the NDA could not be accepted due to issues that are inherent to real-world evidence and external control studies. The Food and Drug Administration (FDA) has ...

Please provide your email address to receive an email when new articles are posted on . Approximately 5,000 people in the United States have Friedreich’s ataxia. Advocates virtually met with ...

Could an anomaly in the developing brain explain motor difficulties occurring decades later in people with rare movement disorders? These are the genetics being investigated by researchers at ...

The National Ataxia Foundation (NAF) is an organization devoted to raising awareness, supporting families, and funding research for Ataxia, a rare and progressive neurological condition that impairs ...

Cerebellum (Latin: "little brain") in orange. Cerebellar means "relating to the cerebellum." Source: 3dMediSphere / Shutterstock Since the 1970s, I've been intrigued by the cerebellum's role in peak ...

“Mild off-road capability” might sound like a marketing gimmick, but it’s actually a useful approach to meet the needs of buyers who want just a bit more ruggedness. The phrase “mild off-road ...

Not only can such devices be awkward, many people also perceive them as being stigmatizing. That's where the GyroPack backpack comes in. The device was first developed by Prof. Heike Vallery's team at ...

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

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 ...

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 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 ...